Showing posts with label FDA approval. Show all posts
Showing posts with label FDA approval. Show all posts

Friday, January 15, 2021

FDA Approves Qdolo (tramadol hydrochloride) Oral Solution for the Management of Severe Pain

In continuation of my update on tramadol 

Bioscience, LLC, a specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved Qdolo™ (tramadol hydrochloride) Oral Solution 5mg/1mL C-IV, an opioid agonist indicated in adults for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate.




According to the Centers for Disease Control and Prevention, 50 million adults in the United States have chronic daily pain, with 19.6 million adults experiencing high-impact chronic pain that interferes with daily life or work activities. A May, 2019 U.S. Department of Health and Human Services report on pain management best practices emphasized the need for an "individualized, patient-centered" approach for the treatment of pain.1

"As an oral liquid, Qdolo gives physicians flexibility to titrate dosing precisely according to individual patients' needs," said Jeff Bryant, President and CEO of Athena Bioscience. "In addition, for patients with swallowing disfunction (dysphagia) or who simply have trouble swallowing pills, Qdolo provides an essential alternative to other forms of tramadol." Qdolo has a patent pending.

https://en.wikipedia.org/wiki/Tramadol


Thursday, January 14, 2021

FDA Approves Onureg (azacitidine tablets) as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia

Bristol Myers Squibb (NYSE: BMY) announced that the U.S. Food and Drug Administration (FDA) has approved Onureg (azacitidine 300 mg tablets, CC-486) for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy.  AML is one of the most common acute leukemias in adults.






The approval is based on results from the pivotal Phase 3 QUAZAR® AML-001 study in which treatment with Onureg resulted in a statistically significant and clinically meaningful improvement in overall survival (OS), the study’s primary endpoint, of nearly 10 months compared to placebo. Median OS from time of randomization was greater than two years (24.7 months; 95% Confidence Interval [CI]: 18.7 to 30.5) among patients who received Onureg compared to 14.8 months (95% CI: 11.7 to 17.6) among patients receiving placebo (Hazard Ratio [HR]: 0.69, 95% CI: 0.55 to 0.86; p=0.0009). Onureg was continued until disease progression or unacceptable toxicity. Onureg has warnings and precautions for risks of substitution with other azacitidine products, myelosuppression, increased early mortality in patients with myelodysplastic syndromes (MDS) and embryo-fetal toxicity. Due to substantial differences in the pharmacokinetic parameters, Onureg should not be substituted for intravenous or subcutaneous azacitidine as it may result in a fatal adverse reaction. New or worsening Grade 3 or 4 neutropenia and thrombocytopenia occurred in 49% and 22% of patients who received Onureg, respectively. Febrile neutropenia occurred in 12% of patients. Complete blood counts should be monitored, dosing should be modified as recommended and standard supportive care should be provided if myelosuppression occurs. Enrollment was discontinued early in the study AZA-MDS-003 due to a higher incidence of early fatal and/or serious adverse reactions in the Onureg arm compared with the placebo arm. Treatment of MDS with Onureg is not recommended outside of controlled trials. Onureg can cause fetal harm when administered to a pregnant woman.

https://en.wikipedia.org/wiki/Azacitidine

Friday, April 17, 2020

A novel pill to treat bleeding from uterine fibroids aims for FDA approval


In continuation of my update on elagolix 
Elagolix.svg

A new drug called elagolix cut blood loss by half over six months in the overwhelming majority of  who participated in two clinical trials published Wednesday in the New England Journal of Medicine.
Elagolix, being developed by AbbVie, was approved by the U.S. Food and Drug Administration in 2018 under the brand name Orilissa to reduce the pain of endometriosis—another common, debilitating female disorder.
The fibroid studies, conducted at 77 sites in the United States and Canada, were led by William D. Schlaff, the chair of obstetrics and gynecology at Thomas Jefferson University in Philadelphia. The results have been submitted to the FDA, which is expected to issue its decision in the first half of 2020.
Elagolix works by suppressing the gonadotropin  and the ovarian sex hormones, estrogen and progesterone. In effect, it throws women into a temporary menopause.
Because this suppression can cause , the researchers gave a subset of women low doses of sex hormones along with elagolix. Of those 395 women, about 70% cut their blood loss by half without suffering more bone thinning than women taking a placebo. The "add-back" hormones also reduced menopausal side effects such as hot flashes and night sweats, although these remained common.
"As a clinician working with patients like this for 40 years, I think this is a valuable clinical tool," Schlaff said of elagolix and add-back hormones. "It's oral, the effect is fast onset, and the side-effect profile is tolerable."
The brand name and pricing that would be used if the FDA approves this use of elagolix have not yet been established for the drug, an AbbVie spokesperson said. The list price for a four-week supply of Orilissa is $907.39.
An estimated 80% of women approaching menopause have fibroids—muscular growths in the uterine wall. Half of those women will develop symptoms, primarily heavy menstrual bleeding, which can lead to a blood iron deficiency. Charlotte Owens, medical director at AbbVie, noted that African American women have a higher risk for  and often develop more severe symptoms than Caucasian women.
After menopause, when the ovaries shut down, menstrual bleeding stops, but many women find the transitional bleeding so troublesome that they seek treatment.
Current options—including drugs that target hormones, procedures that destroy the uterine lining or surgical removal of the fibroids or entire uterus—all have drawbacks. A device called an electric morcellator, which minces fibroids and removes the tissue through tiny incisions, has been largely abandoned because in rare cases it can disseminate an undetected uterine cancer. Philadelphia cardiac surgeon Hooman Noorchashm and his late wife, anesthesiologist Amy Reed, campaigned for a ban on morcellators after her cancer was spread during a hysterectomy.
Existing gonadotropin hormone-suppressing drugs, including one approved in 1989 that Schlaff helped to test in patients, have to be given as injections and take up to two weeks to begin working.
Other companies besides AbbVie have been working to tap the potentially huge market of women with fibroid-related bleeding. A few years ago, Allergan seemed to be in the lead with ulipristal acetate, brand name Esmya, which was already approved in Europe. But after European regulators initiated an investigation into whether the drug led to liver damage in some patients, the FDA declined to approve it.
Myovant Sciences' relugolix, which reduced blood loss in nearly three-quarters of patients in the second of two late-stage clinical trials, is also seeking approval for the  in combination with add-back hormone medications.
https://en.wikipedia.org/wiki/Elagolix

Thursday, March 26, 2020

FDA Approves Valtoco (diazepam nasal spray) as a Seizure Rescue Treatment

Image result for Valtoco


Neurelis, Inc.,  announced that the U.S. Food and Drug Administration (FDA) has approved Valtoco (diazepam nasal spray) as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in people with epilepsy 6 years of age and older. The unique formulation of Valtoco incorporates Intravail® for consistent and reliable absorption.

“Cluster or acute repetitive seizures are challenging to treat and highly disruptive in the lives of people with epilepsy,” said Neurelis President and CEO Craig Chambliss. “Valtoco was developed to provide an effective combination of reliability, safety and tolerability in a ready-to-use nasal spray. This is a defining moment for Neurelis as Valtoco is our first FDA-approved product. We are excited that we can now offer this treatment option to patients and provide additional support to the epilepsy community.”
Chambliss added that Valtoco was also granted seven years of Orphan Drug Exclusivity by the FDA Office of Orphan Products Development.
Valtoco is a proprietary formulation of diazepam incorporating the Science of Intravail. Intravail transmucosal absorption enhancement technology enables the non-invasive delivery of a broad range of protein, peptide and small molecule drugs. In the United States, there are over 3.4 million people with epilepsy, with approximately 200,000 new patients diagnosed each year. Despite the availability of chronic, daily oral medications to control epilepsy, a significant number of these patients continue to experience seizures. Of these uncontrolled patients, as many as 170,000 are at risk for episodes of frequent seizure activity, also known as cluster or acute repetitive seizures, representing a significant unmet need in the epilepsy community.
“This is an important development in the epilepsy community,” said R. Edward Hogan, MD, Director of the Washington University and Barnes-Jewish Epilepsy Center in St. Louis. “Most seizures that require intervention are treated in an inconvenient manner. To be able to reliably treat seizure activity when and where it happens with a caregiver-administered option like Valtoco is a significant step forward. The availability of Valtoco may positively impact the lives of thousands of people with epilepsy who experience cluster or acute repetitive seizures and their care partners.”
In a long-term, open-label, repeat dose, clinical trial, the safety of Valtoco was evaluated: over 130 patients were enrolled and more than 2,000 seizures were treated. The clinical trial included patients aged 6 and above. “Until recently, approved treatment outside of medical care settings was only available as a rectally administered medication,” Dr. Hogan said. “The FDA approval of diazepam nasal spray is a significant advancement for the epilepsy community.”
Enrique Carrazana, MD, Chief Scientific Officer for Neurelis, notes that Valtoco was generally safe and well tolerated during clinical studies. The most common adverse reactions (at least 4%) were somnolence, headache, and nasal discomfort.
Jacqueline A. French, MD, professor in the Department of Neurology at NYU Langone Health’s Comprehensive Epilepsy Center and Chief Medical & Innovation Officer for the Epilepsy Foundation, commented, “One of the goals of rescue therapy is to treat seizure clusters, recognized as medical emergencies, before negative consequences may be experienced. These consequences may include injury and seizure progression to status epilepticus. Having a seizure rescue treatment that is generally safe, reliable and ready-to-use is very empowering. We encourage all epilepsy patients to work with their doctors to make sure they have a seizure rescue treatment plan in place.”
Indication
Valtoco (diazepam nasal spray) is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (ie, seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 6 years of age and older.
https://www.chemdiv.com/valtoco-is-approved-by-fda/

Monday, December 23, 2019

Dova Pharmaceuticals Announces FDA Approval of Doptelet (avatrombopag) for Treatment of Chronic Immune Thrombocytopenia (ITP)


In continuation of my update on avatrombopag
Avatrombopag.svg

Dova Pharmaceuticals, Inc.  a pharmaceutical company focused on acquiring, developing and commercializing drug candidates for diseases where there is a high unmet need, today announced the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) that expands the use of Doptelet (avatrombopag) to include the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. 
Doptelet is also FDA-approved for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.  Earlier this week, Dova announced the marketing authorization granted by the European Commission for Doptelet for the treatment of severe thrombocytopenia in adult patients with CLD who are scheduled to undergo an invasive procedure.
“Dova is pleased to provide Doptelet to patients and physicians in the United States for the treatment of chronic ITP in adult patients who have had an insufficient response to a previous treatment,” said Dr. David Zaccardelli, president and CEO of Dova. “In addition to offering patients with ITP a new treatment option, we expect Doptelet will also address an important unmet medical need in the market.  We sincerely thank the patients and dedicated researchers who participated in our clinical program as well as FDA for their collaboration during the review of this application.”
Doptelet is an oral, thrombopoietin receptor agonist (TPO-RA) administered with food.  In the pivotal Phase 3 study, Doptelet administration resulted in a platelet count of at least 50,000 per µL at day eight of therapy in the majority of patients, with efficacy superior to placebo in maintaining platelet counts in the target range during the 6-month treatment period.  Additional supportive efficacy data for the ITP sNDA were provided by two Phase 2 ITP clinical trials, as well as two Phase 3 trials for the treatment of thrombocytopenia in patients with CLD. 
Safety data for 128 patients with ITP, and more than 1,000 subjects treated across 24 studies in the Doptelet clinical development program across multiple indications, support the safety and tolerability of Doptelet. 
“ITP patients should work with their clinician to choose a therapy that supports their lifestyle and aims to achieve the best possible result to treat their ITP. That’s why having additional treatment options are so important,” said Caroline Kruse, president and CEO of the Platelet Disorder Support Association, a patient advocacy organization dedicated to ITP patients. “We are thrilled to have a new, oral TPO-RA available for adult patients with ITP.  Every new treatment provides more choices and new hope to our community.”
Dova is committed to enabling patient access to Doptelet.  Doptelet will be priced similarly to other TPO-RAs used to treat ITP, and Dova will continue to offer Patient Assistance and Co-Pay programs. The commercial launch of Doptelet for ITP is anticipated to occur in mid-July 2019. 
Dova also entered into an expanded partnership in the United States with Salix. Starting on July 1, 2019, in addition to the gastroenterology, colorectal surgery, and proctology segments, Salix will have the exclusive right to co-promote the CLD indication for Doptelet to the hepatology and interventional radiology segments.  Dova will continue to pay Salix a commission based on a percentage of net sales in these specialties, which will be in the mid-thirties beginning on July 1, 2019.  In addition, the co-promotion agreement was extended to September 2023.
Dr. Zaccardelli added, “The expanded partnership with Salix builds additional momentum for Doptelet and enables the Dova team to focus on a successful launch of the ITP indication. As a growing leader in the treatment of thrombocytopenia, we are committed to realizing Doptelet’s significant market opportunity in CLD, ITP and potentially chemotherapy-induced thrombocytopenia (CIT) for which we expect Phase 3 trial top-line results in the first half of 2020.”
https://pubchem.ncbi.nlm.nih.gov/compound/AVATROMBOPAG#section=3D-Conformer
https://en.wikipedia.org/wiki/Avatrombopag


Friday, November 29, 2019

FDA Approves Second Drug, Vyleesi, to Help Women With Low Libido

In continuation of my update on bremelanotide
Bremelanotide structure.svg
The U.S. Food and Drug Administration on Friday gave its approval to Vyleesi, the second medication so far approved to help women with low sexual desire.
In a news release, the FDA said that Vyleesi (bremelanotide) is a drug that would be administered by injection prior to having sex.
It's been specifically approved for premenopausal women with a condition known as acquired, generalized hypoactive sexual desire disorder (HSDD).
"There are women who, for no known reason, have reduced sexual desire that causes marked distress, and who can benefit from safe and effective pharmacologic treatment," said Dr. Hylton Joffe, who directs the FDA's Center for Drug Evaluation and Research's Division of Bone, Reproductive and Urologic Products.
"Today's approval provides women with another treatment option for this condition," Hylton said in the news release.
According to the agency, HSDD is not caused by any medical or psychiatric condition, relationship issues or drug side effects.
Instead, women with HSDD have "previously experienced no problems with sexual desire," the FDA said. "Generalized HSDD refers to HSDD that occurs regardless of the type of sexual activity, situation or partner."
The exact way in which Vyleesi helps stimulate sexual desire remains unclear, but it works on melanocortin receptors on cells, the FDA said.
The drug is injected under the skin of the abdomen or thigh at least 45 minutes prior to a sexual encounter, although the best timeframe for dosing could vary from user to user.
Side effects can occur, the FDA added, and include nausea and vomiting, flushing, injection site reactions and headache. Nausea was especially common, affecting 40% of users in the clinical study that led to approval.
That study involved 1,247 premenopausal women with HSDD who received Vyleesi or a placebo in one of two 24-week trials.
"In these trials, about 25% of patients treated with Vyleesi had an increase of 1.2 or more in their sexual desire score (scored on a range of 1.2 to 6.0, with higher scores indicating greater sexual desire) compared to about 17% of those who took placebo," the FDA noted.
Still, the overall benefit was not large. "There was no difference between treatment groups in the change from the start of the study to end of the study in the number of satisfying sexual events. Vyleesi does not enhance sexual performance," the FDA said.
And there was one other caveat: Vyleesi can hike blood pressure, so people with heart disease or high blood pressure should not take it, the FDA said.
Vyleesi should also not be taken by anyone who is also taking the drug naltrexone, used to combat opioid dependency, because Vyleesi reduces naltrexone's effectiveness.
Vyleesi is not the first drug approved to enhance flagging libido in women. In 2015 the FDA approved Addyi (flibanserin) for the purpose, but the drug did not become widely used because it cannot be taken with alcohol and only certain certified health care providers are allowed to prescribe it.
According to CNN, Vyleesi's maker, AMAG Pharmaceuticals, said the new drug will not be available until September, and pricing and reimbursement have yet to be determined.
One expert in female sexual health said it remains to be seen how widely Vyleesi will be used.
"Female sexual dysfunction is more complicated in some ways than male sexual dysfunction, so it's more difficult to treat," Dr. Nicole Cirino, co-director of the Menopause and Sexual Therapy Clinic at Oregon Health and Science University's Center for Women's Health, told CNN. She had no role in Vyleesi's development.
Cirini suspects Vyleesi probably will not be the first option women with HSDD turn to, but it might prove a useful adjunct to standard psychotherapy and Addyi.
Vyleesi, like Addyi, probably won't be overprescribed, Cirino added. When Addyi was introduced, there were concerns "that doctors would just be prescribing this medication to anybody that came in saying that they were having an issue with their libido," she said. "And I think we have to give physicians more credit than that. In fact, that didn't happen at all."
Still, Vyleesi could help some women, Cirino said

https://en.wikipedia.org/wiki/Bremelanotide

Thursday, November 28, 2019

FDA Approves Secuado (asenapine) Transdermal System for the Treatment of Adults with Schizophrenia

Noven Pharmaceuticals, Inc., a wholly-owned subsidiary of Hisamitsu Pharmaceutical Co., Inc., today announced the U.S. Food and Drug Administration (FDA) has approved Secuado (asenapine see below pic)) transdermal system, the first-and-only transdermal patch formulation for the treatment of adults with schizophrenia.

Skeletal formula of asenapine

“As people living with schizophrenia cycle through treatments their therapeutic options narrow, leaving them and their caregivers looking for new treatment options,” said Leslie Citrome, M.D., M.P.H., Clinical Professor of Psychiatry and Behavioral Sciences, New York Medical College. “In addition to offering a new delivery option, transdermal patches can also provide caretakers and healthcare providers with a non-intrusive, visual confirmation that a treatment is being utilized.”
The once-daily transdermal drug delivery system (TDDS) provides sustained concentrations during wear time (24 hours)2 of the atypical antipsychotic drug asenapine, a well-established treatment for schizophrenia. A transdermal patch may help to mitigate some of the challenges patients face with the management of their schizophrenia.2
“There is an enormous unmet need for new types of schizophrenia treatments, and Noven is committed to giving people living with this devastating disease and their family members new options that may help them effectively manage their symptoms,” said Dr. Naruhito Higo, Chairman and Chief Executive Officer, Noven Pharmaceuticals, Inc. “We commend the FDA on the approval of Secuado and look forward to bringing it to market in the U.S. as soon as possible so people living with schizophrenia have a transdermal delivery option for asenapine treatment.”
In the international, Phase 3, double-blind, placebo-controlled study, Secuado achieved the primary endpoint of statistically significant improvement from baseline in the change of the total Positive and Negative Syndrome Scale (PANSS) compared to placebo at week six. Efficacy and safety were assessed during the six-week treatment period in 616 adults living with schizophrenia. Additionally, Secuado demonstrated statistically significant improvement in Clinical Global Impression-Severity (CGI-S) scores, the key secondary endpoint of the Phase 3 study.
The systemic safety profile of Secuado was consistent with what is known for sublingual asenapine.1 The most commonly observed adverse reactions were extrapyramidal disorder, application site reaction, and weight gain.1
What is Secuado?
Secuado is a prescription medicine used to treat adults with schizophrenia. Secuado is a transdermal system (patch) you apply to your skin. It is not known if Secuado is safe and effective in children less than 18 years of age with schizophrenia.
IMPORTANT SAFETY INFORMATION
Secuado may cause serious side effects, including:


  • Increased risk of death in elderly people with dementia-related psychosis. Medicines like Secuado can raise the risk of death in elderly people who have lost touch with reality (psychosis) due to confusion and memory loss (dementia). Secuado is not approved for the treatment of people with dementia-related psychosis.
  • Stroke (cerebrovascular problems) in elderly people with dementia-related psychosis that can lead to death.
  • Neuroleptic Malignant Syndrome (NMS): a serious condition that can lead to death. Immediately remove the patch. Call your healthcare provider or go to the nearest hospital emergency room right away if you have some or all of the following: high fever, confusion, stiff muscles, increased sweating and changes in your breathing, heart rate and blood pressure.
  • Uncontrolled body movements (tardive dyskinesia). Secuado may cause movements that you cannot control in your face, tongue, or other body parts. Tardive dyskinesia may not go away, even if you stop taking Secuado. Tardive dyskinesia may also start after you stop taking Secuado.
  • Problems with your metabolism such as:
    • High blood sugar (hyperglycemia) and diabetes. Increases in blood sugar can happen in some people who take Secuado.
      Call your healthcare provider if you have any of these symptoms of high blood sugar during treatment with Secuado:
      • Feel very thirsty or very hungry
      • Feel sick to your stomach
      • Feel weak or tired
      • Need to urinate more than usual
      • Feel confused, or your breath smells fruity
    • Increased fat levels (cholesterol and triglycerides) in your blood
    • Weight gain. You and your healthcare provider should check your weight regularly during treatment with Secuado.
  • Allergic reactions. You may observe rash, decreased blood pressure or a fast heart rate.
  • Decreased blood pressure (orthostatic hypotension). You may feel lightheaded or faint when you rise too quickly from a sitting or lying position.
  • Falls. Secuado may make you sleepy or dizzy, may cause a decrease in your blood pressure when changing position, and can slow your thinking which may lead to falls.
  • Low white blood cell count. Your healthcare provider may do blood tests during the first few months of treatment with Secuado.
  • Irregular heartbeat or a heartbeat that does not feel normal (QT prolongation)
  • Increased prolactin levels in your blood (hyperprolactinemia). Your healthcare provider may do blood tests to check your prolactin levels during treatment with Secuado.
  • Seizures (convulsions)
  • Impaired thinking and motor skills. Use caution when operating heavy machinery when using Secuado.
  • Problems controlling your body temperature so that you feel too warm
  • Difficulty swallowing
  • External heat. Avoid exposing Secuado to direct external heat sources such as hair dryers, heating pads, electric blankets, heated water beds, etc.
  • Application site reactions. Increased skin irritation may occur if Secuado is applied for a longer period than instructed or if the same application site is used repeatedly. Use a different application site each day to decrease skin reactions. If skin reactions continue or spread beyond the application site, tell your healthcare provider. Symptoms of application site reactions may include:
    • Redness
    • Itching
    • Irritation
    • Pimple-like raised skin
    • Pain of the skin
    • Swelling
  • More 
Ref : https://en.wikipedia.org/wiki/Asenapine



Wednesday, November 27, 2019

FDA Approves Nayzilam (midazolam) Nasal Spray to Treat Seizure Clusters



  Thumb




UCB announced   that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application for the company’s newest anti-epileptic drug (AED) Nayzilam (midazolam) nasal spray CIV, a benzodiazepine indicated for the acute treatment of intermittent, stereotypic episodes of  frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. Nayzilam now provides patients and caregivers with the first and only FDA-approved nasal option for treating seizure clusters.  


It is estimated that more than 150,000 people in the U.S. with uncontrolled epilepsy also experience seizure clusters.2 Rescue treatment of seizure clusters is critical because when left untreated, seizure clusters can increase the risk of physical injury, neurological damage, prolonged seizures, and status epilepticus. Despite the impact of seizure clusters, many diagnosed patients may go untreated because currently available treatment options are not preferred.



Nayzilam is a short-term treatment for seizure clusters in patients with epilepsy. The nasal spray is designed as a single-use treatment that can be carried with a patient. Nayzilam allows for administration by a non-healthcare professional in patients actively seizing when and where a seizure cluster occurs. Nayzilam can provide value to patients who are experiencing these disruptive seizures.



“As global leaders in epilepsy, the approval of Nayzilam complements our already strong epilepsy portfolio, improving our ability to provide value to people living with poorly controlled seizures, and builds on our passion and expertise in this field. We are pleased to expand and diversify the solutions we can offer to the epilepsy community, providing an innovative and differentiated solution to help support management of seizure clusters,” said Jean-Christophe Tellier, Chief Executive Officer, UCB.



Nayzilam is the first new medication approved to treat seizure clusters in more than 20 years in the U.S. Its nasal delivery could provide significant value to patients who currently have limited treatment options.



“When a patient experiences seizure clusters, there is often significant impact on their overall quality of life, in addition to posing greater risks for increased emergency department related hospitalizations and more serious seizure emergencies,” said Dr. Steven S. Chung, MD, Executive Director and Program Chair of the Neuroscience Institute and Director of the Epilepsy Program at Banner – University Medical Center. “Further, as a neurologist specializing in epilepsy, treating seizure clusters today presents a challenging barrier for many patients. The availability of a new treatment option, such as Nayzilam, has potential to help improve the lives of patients and their families by providing another option for rescue care.”


https://www.drugbank.ca/drugs/DB00683

Thursday, October 10, 2019

FDA Approves Victoza (liraglutide) for the Treatment of Pediatric Patients 10 Years or Older with Type 2 Diabetes


In continuation of my update on Victoza (liraglutide)
The U.S. Food and Drug Administration   approved Victoza (liraglutide) injection for treatment of pediatric patients 10 years or older with type 2 diabetes. Victoza is the first non-insulin drug approved to treat type 2 diabetes in pediatric patients since metformin was approved for pediatric use in 2000. Victoza has been approved to treat adult patients with type 2 diabetes since 2010.
“The FDA encourages drugs to be made available to the widest number of patients possible when there is evidence of safety and efficacy,” said Lisa Yanoff, M.D, acting director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research. “Victoza has now been shown to improve blood sugar control in pediatric patients with type 2 diabetes. The expanded indication provides an additional treatment option at a time when an increasing number of children are being diagnosed with this disease.”
Type 2 diabetes is the most common form of diabetes, occurring when the pancreas cannot make enough insulin to keep blood sugar at normal levels. Although type 2 diabetes primarily occurs in patients over the age of 45, the prevalence rate among younger patients has been rising dramatically over the past couple of decades. The Diabetes Report Card published by the U.S. Centers for Disease Control and Prevention estimates that more than 5,000 new cases of type 2 diabetes are diagnosed each year among U.S. youth younger than age 20.
Victoza improves blood sugar levels by creating the same effects in the body as the glucagon-like peptide (GLP-1) receptor protein in the pancreas. GLP-1 is often found in insufficient levels in type 2 diabetes patients. Like GLP-1, Victoza slows digestion, prevents the liver from making too much glucose (a simple sugar), and helps the pancreas produce more insulin when needed. As noted on the label, Victoza is not a substitute for insulin and is not indicated for patients with type 1 diabetes or those with diabetic ketoacidosis, a condition associated with diabetes where the body breaks down fat too quickly because there is inadequate insulin or none at all. Victoza is also indicated to reduce the risk of major adverse cardiovascular events in adults with type 2 diabetes and established cardiovascular disease; however, its effect on major adverse cardiovascular events in pediatrics was not studied and it is not indicated for this use in children.
The efficacy and safety of Victoza for reducing blood sugar in patients with type 2 diabetes was studied in several placebo-controlled trials in adults and one placebo-controlled trial with 134 pediatric patients 10 years and older for more than 26 weeks. Approximately 64% of patients in the pediatric study had a reduction in their hemoglobin A1c (HbA1c) below 7% while on Victoza, compared to only 37% who achieved these results with the placebo. HbA1c is a blood test that is routinely performed to evaluate how well a patient’s diabetes is controlled, and a lower number indicates better control of the disease. These results occurred regardless of whether the patient also took insulin at the same time. Adult patients who took Victoza with insulin or other drugs that increase the amount of insulin the body makes (e.g., sulfonylurea) may have an increased risk of hypoglycemia (low blood sugar). Meanwhile, pediatric patients 10 years and older taking Victoza had a higher risk of hypoglycemia regardless of whether they took other therapies for diabetes.
The prescribing information for Victoza includes a Boxed Warning to advise health care professionals and patients about the increased risk of thyroid C-cell tumors. For this reason, patients who have had, or have family members who have ever had medullary thyroid carcinoma (MTC) should not use Victoza, nor should patients who have an endocrine system condition called multiple endocrine neoplasia syndrome type 2 (MEN 2). In addition, people who have a prior serious hypersensitivity reaction to Victoza or any of the product components should not use Victoza. Victoza also carries warnings about pancreatitis, Victoza pen sharing, hypoglycemia when used in conjunction with certain other drugs known to cause hypoglycemia including insulin and sulfonylurea, renal impairment or kidney failure, hypersensitivity and acute gallbladder disease. The most common side effects are nausea, diarrhea, vomiting, decreased appetite, indigestion and constipation.

https://en.wikipedia.org/wiki/Liraglutide

Wednesday, October 9, 2019

FDA Approves Symdeko (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in the CFTR Gene

In continuation of my update on tezacafto
200px   Tezacaftor 
                                            Ivacaftor and lumacaftor.svg
                                                                                             Lumacaftor/ivacaftor
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)  announced the U.S. Food and Drug Administration (FDA) approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Symdeko. It was previously approved by the FDA for use in patients with cystic fibrosis 12 years and older with two copies of the F508del mutation or one copy of a responsive mutation in the U.S. An additional dosage strength of Symdeko tablets is now available (tezacaftor 50 mg/ivacaftor 75 mg and ivacaftor 75 mg) in connection with this approval.
“Today’s expanded approval of Symdeko in children ages 6 through 11 is an important step in our efforts to continue to bring treatment options to the youngest patients possible and importantly brings us closer to our goal of developing medicines for all people living with CF,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex.
Vertex completed a 24-week Phase 3 open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of tezacaftor/ivacaftor and ivacaftor in children ages 6 through 11 years in the U.S. and Canada. The regimen was generally well tolerated, and safety data were similar to what was observed in previous studies of patients aged 12 years and older. The full data from this study will be published later this year.
“We’ve seen the clinical impact of Symdeko in people with CF aged 12 years and above, and this approval marks a crucial milestone for patients ages 6 through 11 years who may benefit from CFTR modulation, enabling us to treat the basic defect in CF at an earlier stage of disease,” said Seth Walker, M.D., University Hospitals of Cleveland, Cleveland Medical Center, Rainbow Babies and Children’s Hospital. “Symdeko is an important treatment option for eligible people with CF who either never started or have discontinued another CFTR modulator.”

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.
About Symdeko (tezacaftor/ivacaftor and ivacaftor) 
Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Symdeko is a combination of tezacaftor and ivacaftor. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.


U.S. INDICATION FOR SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) tablets 
Symdeko is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with Symdeko. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if Symdeko is safe and effective in children under 6 years of age.


https://en.wikipedia.org/wiki/Tezacaftor
https://en.wikipedia.org/wiki/Lumacaftor/ivacaftor

Tuesday, October 8, 2019

FDA Approves Gattex (teduglutide) for Children 1 Year of Age and Older With Short Bowel Syndrome (SBS)

In continuation of my update on Teduglutide

Teduglutide.png

Takeda Pharmaceuticals, U.S.A., Inc. (“Takeda”), announced  that the U.S. Food and Drug Administration (FDA) approved extending the indication of Gattex(teduglutide) for injection to pediatric patients 1 year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous (IV) feeding (parenteral support).
In children, SBS is a life-threatening, chronic, and rare malabsorption disorder resulting from surgical removal of a large portion of the intestine, which is typically due to congenital or acquired conditions of the newborn or trauma.2-4 Children with SBS are unable to absorb enough nutrients and fluids from what they eat and drink alone.2 A goal of SBS treatment is to restore the remaining intestine’s ability to absorb nutrients and reduce long-term dependence on parenteral support (PS).
“As a pediatric gastroenterologist, one of my main treatment goals for children with SBS is to reduce their dependency on parenteral support,” said Beth Carter, MD, Medical Director of Intestinal Rehabilitation and Nutrition Support, Children’s Hospital Los Angeles. “I’m pleased that patients have access to a medication that may help them reach that goal.”
Gattex is the first and only medicine that mimics naturally occurring glucagon-like peptide-2 (GLP-2), which helps the remaining intestine absorb more nutrients.1 In a pharmacodynamic study in adults, Gattex was shown to improve the amount of fluids absorbed by the intestines.
“Addressing high unmet needs of patients with complex and debilitating gastrointestinal (GI) conditions is a focus of Takeda’s work,” said Andrew Grimm, Global Clinical Development Lead, Takeda. “As the first U.S.-approved therapy in pediatric SBS patients dependent on PS that improves absorption, Gattex offers these patients new hope to reduce PS requirements and the potential for PS independence. This approval underscores Takeda’s commitment to patients with rare and devastating GI conditions like SBS.”
In a 24-week pediatric study, Gattex helped reduce the volume of daily PS required and time spent administering PS. Some children even achieved complete freedom from PS.1 Fifty- nine pediatric patients with SBS aged 1 year through 17 years chose whether to receive Gattex or standard of care (SOC). Patients who chose to receive Gattex treatment were subsequently randomized in a double-blind manner to 0.025 mg/kg/day (n=24) or 0.05 mg/kg/day (n=26), while 9 patients enrolled in the SOC arm. The recommended dosage of Gattex is 0.05 mg/kg/day. Randomization to the Gattex dose groups was stratified by age.
At the end of the 24-week study, 69% of patients (18/26) who took Gattex 0.05 mg/kg each day reduced PS volume by 20% or more. Based on patient-diary data, patients who received Gattex 0.05 mg/kg/day experienced a 42% mean reduction in PS volume (mL/kg/day) from baseline (-23 mL/kg/day from baseline). At week 24, 38% of patients (10/26) were able to reduce PS infusion by at least 1 day per week. Patients reduced their PS infusion time by 3 hours per day on average compared to baseline. In addition, during this study 3 out of 26 (12%) children who received Gattex 0.05 mg/kg/day completely weaned off PS.
Gattex has a demonstrated safety profile that is similar overall in pediatric and adult patients. The most common adverse reactions (≥10%) seen in adult patients treated with Gattex in clinical trials were abdominal pain, nausea, upper respiratory tract infection, abdominal distension, injection site reaction, vomiting, fluid overload, and hypersensitivity
https://en.wikipedia.org/wiki/Teduglutide