Thursday, March 22, 2018

Sorrento Therapeutics Subsidiary, Scilex, Receives FDA Approval for Non-Opioid ZTlido (lidocaine topical system) 1.8% for Post-Herpetic Neuralgia Pain

In continuation of my update on lidocaine


Sorrento Therapeutics, Inc. , received approval from the U.S. Food and Drug Administration (FDA) for ZTlido (lidocaine topical system) 1.8%. ZTlido is indicated for the relief of pain associated with post-herpetic neuralgia (PHN), also referred to as post-shingles pain. ZTlido is a major advancement in analgesics because of its proprietary adhesion technology demonstrating 12-hour wear with efficient lidocaine delivery, even during exercise.
“ZTlido was designed to solve a problem that is commonly reported with transdermal/topical patches: they don’t stay on. Based on the adhesion study results with ZTlido, we believe that ZTlido product will be welcomed by healthcare providers, patients and payers who are looking for an effective and efficient, local pain treatment,” said Dr. Henry Ji, Chairman and CEO of Sorrento and Scilex. “We also intend to explore the expansion of ZTlido into additional indications and the underlining platform technology of ZTlido for other active pharmaceutical ingredients (APIs) and combinations of APIs. As demonstrated by the NDA approval for ZTlido, our team successfully executed on our development plan for the product and now, looks forward to executing on our commercial and strategic alliance plans as well.”
“Topical lidocaine is an important option for healthcare providers to have in their armamentarium for treating PHN, a difficult-to-treat neuropathic pain,” stated Dr. Jeff Gudin, Director, Pain Management and Palliative Care, Englewood Hospital and Medical Center. “The Centers for Disease Control and Prevention’s guideline of non-opioid treatments for chronic pain recognizes topical lidocaine as an alternative first-line therapy. ZTlido now offers providers and patients this option.”
ZTlido’s anhydrous topical system is based on a novel technology that is designed to achieve superior adhesion and drug delivery efficiency. ZTlido only requires 36 mg/topical system versus 700 mg/patch of Lidoderm® (lidocaine patch 5%), the US reference product, to achieve the same therapeutic dose of drug. The safety and efficacy of ZTlido was bridged to Lidoderm in comparative pharmacokinetic studies that demonstrated bioequivalence between products.
According to an FDA report of the product quality of transdermal drug delivery systems, adhesion was the most widely reported quality defect of transdermal patches.¹ With a clear need for improved patch adhesion systems, ZTlido was specifically designed to maintain optimum skin contact throughout the 12-hour administration period. Adhesion is critical to the safety and efficacy quality of a topical system. Simply, the topical system must be in contact with skin to deliver the drug. ZTlido adhesion performance was demonstrated in a clinical study in fifty-four (54) healthy volunteers where forty-seven (47) subjects (87%) had adhesion scores of 0 (≥ 90% adhered; essentially no lift off the skin) for all evaluations performed every 3 hours during the 12 hours of administration, and seven (7) subjects (13%) had adhesion scores of 1 (≥ 75% to < 90% adhered; some edges only lifting off the skin) for at least one evaluation, and no subjects had scores of 2 or greater (< 75% adhered). In the same study 91% (49) of the subjects presented with a score of 0 at the end of the 12-hour administration period. The remaining 5 subjects had a score of 1.
In a separate Phase 1 comparative adhesion study in normal healthy subjects (n=44), ZTlido demonstrated superior adhesion (p <0.0001) to Lidoderm at 3 hours that improved over the 12-hour administration period.
According to recent IMS data, more than 100 million prescription lidocaine patches were sold in the US in 2017. Sorrento intends to have Scilex complete the final steps necessary to commercial launch of ZTlido in the US with the objective to make the product commercially available to patients sometime in 2018.

Wednesday, March 21, 2018

Lilly Receives Additional FDA Approval for Verzenio (abemaciclib), as Initial Treatment for Advanced Breast Cancer

In continuation of my update on Abemaciclib


Eli Lilly and Company  announced the U.S. Food and Drug Administration (FDA) has approval of  Verzenio (abemaciclib) in combination with an aromatase inhibitor (AI) as initial endocrine-based therapy for the treatment of postmenopausal women with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer. This additional FDA approval marks the third indication for Verzenio within five months. In September 2017, Verzenio became the first and only cyclin-dependent kinase (CDK)4 & 6 inhibitor approved in combination and as a single agent in metastatic breast cancer. Specifically, Verzenio was approved for use in combination with fulvestrant for the treatment of women with HR+, HER2- advanced or metastatic breast cancer with disease progression following endocrine therapy, and as monotherapy for the treatment of adult patients with HR+, HER2- advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting.

The recommended dose of Verzenio in combination with an AI is 150 mg orally twice daily, continued until disease progression or unacceptable toxicity. Verzenio is available in four tablet strengths (200 mg, 150 mg, 100 mg, and 50 mg).
This approval of Verzenio as initial therapy in combination with an AI is based on the efficacy and safety demonstrated in the pivotal MONARCH 3 clinical trial. MONARCH 3 is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating Verzenio in combination with an AI as initial endocrine-based therapy that enrolled 493 postmenopausal women with HR+, HER2- advanced breast cancer who had no prior systemic treatment for advanced disease. In patients who received neoadjuvant/adjuvant endocrine therapy, a disease-free interval of more than 12 months since completion of endocrine therapy was required. This Verzenio new drug application was given Priority Review as part of the FDA's Expedited Programs for Serious Conditions, a program used for therapies that address an unmet medical need in the treatment of serious or life-threatening conditions, such as metastatic breast cancer. Verzenio was also granted Breakthrough Therapy Designation in 2015 based on the Phase 1 JPBA trial.
In MONARCH 3, Verzenio dosed orally at 150 mg twice daily on a continuous schedule with an AI demonstrated a greater than 28-month median progression-free survival (PFS) in patients who received initial endocrine-based therapy for metastatic disease (28.2 months [95% CI: 23.5-NR] vs 14.8 months [95% CI: 11.2-19.2] with placebo plus an AI [HR: 0.54; 95% CI: 0.418-0.698, P <0.0001]). In patients with measurable disease who received Verzenio plus an AI (n=267), an objective response rate of 55.4 percent was achieved (ORR; defined as complete response plus partial response [CR + PR], and based upon confirmed responses; PR defined as ≥30% reduction in target lesions)1 (n=148; 95% CI: 49.5-61.4), with 52.1 percent of patients having achieved a PR (n=139) and 3.4 percent having achieved a CR (n=9).2 In comparison, in the placebo-plus-AI group of patients with measurable disease (n=132), ORR was 40.2 percent (n=53; 95% CI: 31.8-48.5), with all women being partial responders. Median duration of response (DoR) was 27.4 months with Verzenio plus an AI (95% CI: 25.7-NR) versus 17.5 months with placebo plus an AI (95% CI: 11.2-22.2).
"This approval is an important milestone, as it shows that Verzenio plus an aromatase inhibitor substantially reduced tumor size and delayed disease progression in women with HR+, HER2- metastatic breast cancer. Notably, the MONARCH 3 trial included patients with certain concerning clinical characteristics, such as a pattern of disease that spread to the liver," said Joyce O'Shaughnessy, M.D., Celebrating Women Chair in Breast Cancer Research and chair, Breast Cancer Research Program, Baylor University Medical Center, Texas Oncology and U.S. Oncology, Dallas, TX. "This information will help inform treatment decisions for each patient, which can be complicated in advanced breast cancer."
The labeling for Verzenio contains warnings and precautions for diarrhea, neutropenia, hepatotoxicity, venous thromboembolism, and embryofetal toxicity. Instruct patients at the first sign of loose stools to initiate antidiarrheal therapy, increase oral fluids, and notify their healthcare provider. Perform complete blood counts and liver function tests prior to the start of Verzenio treatment, every two weeks for the first two months, monthly for the next two months, and as clinically indicated. Based on results, Verzenio may require dose modification. Monitor patients for signs and symptoms of thrombosis and pulmonary embolism and treat as medically appropriate. Advise patients of potential risk to a fetus and to use effective contraception. See full Prescribing Information for further management instructions. The most common adverse reactions in the MONARCH 1, 2, and 3 trials (all grades, ≥20%) were diarrhea, neutropenia, nausea, abdominal pain, infections, fatigue, anemia, leukopenia, decreased appetite, vomiting, headache, alopecia, and thrombocytopenia.
"The speed with which our team has been able to work with the FDA to gain approval for this additional Verzenio indication underscores Lilly's commitment to delivering meaningful medicines that can help more people living with advanced breast cancer," said Sue Mahony, Ph.D., senior vice president and president of Lilly Oncology. "Verzenio has now been developed, studied and clinically proven in three key trials to be effective for women with HR+, HER2- metastatic breast cancer – helping to ensure we are providing support to those who need it most."
"For those facing a diagnosis of metastatic breast cancer or learning that their disease has spread further, each new indication and clinical development is critical," said Marc Hurlbert, Ph.D., chairman, Metastatic Breast Cancer Alliance. "Today's news represents continued progress towards helping more people living with this devastating disease."

Tuesday, March 20, 2018

FDA Approves Apadaz (benzhydrocodone and acetaminophen) for the Short-Term Management of Acute Pain

In continuation of my update on Apadaz 

KemPharm, Inc.     announced  the   FDA  approval of  its New Drug Application (NDA) for Apadaz for the short-term (no more than 14 days) management of acute pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. Apadaz is an immediate release (IR) combination of KemPharm’s prodrug, benzhydrocodone, and acetaminophen (APAP).

Benzhydrocodone.svgbenzhydrocodone               Image result for acetaminophen

“The approval of Apadaz is a significant milestone for KemPharm as it creates the opportunity to introduce what we believe is a differentiated product for the short-term management of acute pain,” said Travis Mickle, Ph.D., KemPharm President and Chief Executive Officer. “Based on its unique properties, we firmly believe there is a commercial pathway for Apadaz in what is a very high-volume market. We are excited by the opportunity Apadaz offers to patients and for physicians who now have the option of prescribing a differentiated product.”
“In addition to today’s approval, the U.S. Drug Enforcement Administration (DEA) has indicated that it is their intent to schedule Apadaz as a C-II product and will provide an allocation of the Active Pharmaceutical Ingredient (API) consistent with those scheduling provisions,” added Dr. Mickle. “This prompt decision by the DEA essentially completes the regulatory process with both Agencies and allows us to shift our focus towards the product launch.”

Monday, March 19, 2018

Beetroot may reduce kidney failure risk after heart x-ray, research reveals

Beetroot may reduce the risk of kidney failure in patients having a heart x-ray, according to research led by Queen Mary University of London.
The new research project funded by national charity Heart Research UK will look into whether dietary inorganic nitrate found commonly in beetroot could be used in pill form to prevent one of the most common causes of kidney failure in hospital.
Coronary angiography is a type of x-ray test which is used to look at the coronary arteries in the heart and diagnose a number of heart conditions. It can also help in the planning of procedures to widen narrowed or blocked arteries in the heart.
During angiography, a special dye is injected into the blood so that the blood vessels can be seen. However the dye can cause acute kidney injury, known as contrast induced nephropathy (CIN), which is thought to be in part because it reduces levels of nitric oxide in the kidneys.
Dietary nitrate, found in abundance in vegetables such as beetroot, can increase levels of nitric oxide in the body.
Professor Amrita Ahluwalia, Co-Director and Professor of Vascular Pharmacology at Queen Mary's William Harvey Research Institute, said: "CIN is a serious consequence of coronary angiography, resulting in patients staying longer in hospitals and higher healthcare costs.
"If we find that giving dietary nitrate in capsule form could replace the lost nitric oxide in the kidneys and prevent CIN, the benefits to patients with heart disease would be substantial including reduced rates of kidney damage, less need for treatments such as dialysis and better long term survival."
Prof Ahluwalia with her colleagues Dr Dan Jones (Senior Lecturer in Clinical Trials) and Prof Anthony Mathur (Director of Intervention, Barts Heart Centre) will divide patients into two groups, one group taking nitrate capsules and the other group taking placebo capsules that do not contain nitrate.
Kidney function will then be measured and compared in both groups before the procedure, and two days and three months after to see if dietary nitrate makes a difference.
Barbara Harpham, Chief Executive of Heart Research UK, said: "Our Translational Research Project Grants aim to bridge a gap between laboratory-based scientific research and patient care, helping benefit patients as soon as possible.
"This exciting project has the potential to help reduce the risk of kidney damage and lead to better long-term survival for patients following coronary angiography."
Queen Mary's William Harvey Research Institute is just one recipient of Heart Research UK's Translational Research Project Grants. Awarded since 2009, the national charity based in Leeds has given almost £5m to fund these innovative and pioneering medical research projects across the UK.

Friday, March 16, 2018

Wine polyphenols may be good for oral health

Sipping wine is good for your colon and heart, possibly because of the beverage's abundant and structurally diverse polyphenols. Now researchers report in ACS' Journal of Agricultural and Food Chemistry that wine polyphenols might also be good for your oral health.
Traditionally, some health benefits of polyphenols have been attributed to the fact that these compounds are antioxidants, meaning they likely protect the body from harm caused by free radicals. However, recent work indicates polyphenols might also promote health by actively interacting with bacteria in the gut. That makes sense because plants and fruits produce polyphenols to ward off infection by harmful bacteria and other pathogens. M. Victoria Moreno-Arribas and colleagues wanted to know whether wine and grape polyphenols would also protect teeth and gums, and how this could work on a molecular level.
The researchers checked out the effect of two red wine polyphenols, as well as commercially available grape seed and red wine extracts, on bacteria that stick to teeth and gums and cause dental plaque, cavities and periodontal disease. Working with cells that model gum tissue, they found that the two wine polyphenols in isolation -- caffeic and p-coumaric acids -- were generally better than the total wine extracts at cutting back on the bacteria's ability to stick to the cells. When combined with the Streptococcus dentisani, which is believed to be an oral probiotic, the polyphenols were even better at fending off the pathogenic bacteria. The researchers also showed that metabolites formed when digestion of the polyphenols begins in the mouth might be responsible for some of these effects.​
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Thursday, March 15, 2018

Daffodils may hold secrets of cancer cure

Daffodils - Image Credit: Servickuz / Shutterstock

A natural extract from daffodils may help in treatment of cancer find researchers at the Faculty of Sciences at the ULB led by Denis Lafontaine. The team published their research titled, “The Amaryllidaceae Alkaloid Haemanthamine Binds the Eukaryotic Ribosome to Repress Cancer Cell Growth” in the journal Structure that appeared in their latest issue this week.

The team managed to extract a natural anti-cancer compound from daffodils (known scientifically as Amaryllidaceae Narcissus. This compound is called haemanthamine. They then noted that haemanthamine tends to bind to ribosomes within the cells. Ribosomes are microscopic molecules within the cell that help in the synthesis of proteins. As the cancer cells multiply rapidly and spread the cancer throughout the body they depend on rapid and effective protein synthesis. If these ribosomes can be stopped, the protein synthesis comes to a halt and this stops the cancer growth in its tracks so to speak. In this new study the team found that haemanthamine can block the production of the proteins by blocking the ribosomes. They also act within the nucleolus by stopping the production of ribosomes itself. This whole process activates a chain of events called the “anti-tumour surveillance” that essentially gears up to stop the tumor growth. A protein associated with cancers called the p53 protein also is stabilized as a result and this stops the cancer growth and eliminates the cancer cells.

Daffodils have been known for their anti-cancer properties in folklore for centuries in the Greek and Roman ages. They have been used for their medicinal properties in treatment of inflammatory diseases, malaria, viral infections etc. They contain several alkaloids such as galanthamine, lycorine (LYC), haemanthamine (HAE), tazettine and haemanthamine precursor haemanthidine (HAD). This is the first study that analyzes and actually looks at the mechanism by which daffodils could help treat cancer. Haemanthamine is also found in other plants and plant products such as morphine from opium plant, quinine from cinchona tree and ephedrine from plant Ephedra sinica.
The authors conclude that in their study testing haemanthamine and its precursor haemanthidine, they find that these can stop cancer cell division. The effect may not be only due to inhibition of protein synthesis, they write but also due to the activation of the “nucleolar surveillance” and “stabilization of p53”. “HAE and HAD might prove advantageous in cancer therapy,” they write. More studies are necessary to transform these study findings into real anti cancer drugs available for treatment.
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Wednesday, March 14, 2018

Omega fatty acid supplements may improve ASD symptoms in toddlers born preterm, study shows

In continuation of my update on omega fatty acids
Researchers from Nationwide Children's Hospital have shown that omega fatty acid supplements may improve autism spectrum disorder symptoms in toddlers who were born very preterm (more than 11 weeks early). The study was published recently in the Journal of Nutrition.
"The trial had two goals. First, we wanted to confirm the feasibility of a large study of toddlers born very preterm and exhibiting symptoms often seen with ASD. Second, we wanted to see what the effects of omega fatty acids would be on parent-reported ASD symptoms and related behaviors," says Sarah Keim, Ph.D., lead author on the study and principal investigator in the Center for Biobehavioral Health in The Research Institute at Nationwide Children's.
Dr. Keim and her team conducted a study where 31 toddlers who were born prematurely participated. For 3 months, half of them took a daily dietary supplement that contained a special combination of omega-3 and omega-6 fatty acids, and the other half took a placebo, although families were unaware of which they received to make the study rigorous.
The group that took the daily omega fatty acid supplement exhibited a greater reduction in ASD symptoms than those who took the placebo, according to ratings provided by the children's parents.
"We found clinically significant improvements in ASD symptoms in the treatment group, although the benefits were confined to one measure we used," explains Dr. Keim. "We need to do a larger trial to further understand the potential impacts on a larger group of children."
The researchers suggest that observed benefits of omega fatty acid supplementation could be due to the role of these nutrients in inflammation in the body. ASD is generally considered a neuroinflammatory condition, and influencing inflammation through nutritional supplementation could improve behaviors in children with ASD symptoms.
Researchers hope that by giving omega fatty acids to children early when they first show symptoms and the brain is still actively developing may help them long-term.
"Currently, no medications are available to help children born prematurely with the developmental delays and behavior problems they often experience. For very young children, the medications that physicians sometimes try tend to have many side effects. And we don't know what effect those medications have on brains that are still developing," says Dr. Keim. "If using omega fatty acid supplementation helps, it would have a really huge impact for these kids."
Dr. Keim and her team plan to expand the work in a full-scale trial in the future. They recently received a grant from the National Institutes of Health to study the effect of omega fatty acids in children ages 2-6 year who have ASD.
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Tuesday, March 13, 2018

Gout medication may help improve heart function in adult patients

Researchers at the University of Cincinnati (UC) College of Medicine have shown that probenecid, a drug long used to treat gout, may be able to improve heart function in adult patients who experience heart failure.
The results are published in the Journal of the American Heart Association based off a study of 20 patients at the University of Cincinnati Medical Center.
"We were testing if probenecid was safe for patients," says Jack Rubinstein, MD, associate professor in the Division of Cardiovascular Health and Disease, and corresponding author for the study. "We know that it was very likely to be safe because the medicine had been taken by people of all ages for decades. It has a very strong safety profile. We were quite happily surprised it improved the two main ways in how the heart functions. It improves how the heart contracts and how it relaxes."
The patients were offered probenecid as part of a randomized, double-blind, crossover and placebo-controlled single-center clinical trial. Patients, who averaged 57 years of age, were enrolled during four-week periods between June 2013 and April 2015.
They were required to undergo an echocardiogram, an electrocardiogram and six-minute endurance test along with other assessments, explains Rubinstein, a UC Health cardiologist and member of the UC Heart, Lung and Vascular Institute.
The study's first author is Nathan Robbins, a senior research assistant in the UC College of Medicine, who started out volunteering with Rubinstein in the laboratory examining the echocardiograms of animals treated with probenecid before later being hired to help recruit heart failure patients.
"This is the first time probenecid has been used in heart failure patients and we showed it increases the ejection fraction in patients with heart failure," says Robbins. "It was exciting to be able to see this medicine work from the bench to the bedside."
Rubinstein also partnered with basic scientists led by Sakthivel Sadayappan, PhD, professor in the UC College of Medicine and director of the heart branch of the UC Heart, Lung and Vascular Institute. Sadayappan and his researchers examined probenecid in animal heart cells and found it improved how well the heart uses calcium, an important component in cardiac muscle contraction.
"The medicine works in ways we know about and in ways we don't know about," says Rubinstein. "For the past four or five years we have been figuring out some of the ways the medicine works. We have figured out a lot of them, but there is still a lot we don't know."
Heart failure occurs when the heart pump is not strong enough to move blood throughout the body and meet the body's needs for oxygen, explains Rubinstein. It affects 5.7 million people in the United States, according to the Centers for Disease Control and Prevention.
"The repercussions are potentially significant--if we are able to confirm this experiment in larger studies with longer-term follow up--this could present a new way of treating heart failure for which there are limited medical therapies available," says Rubinstein.
"Left ventricular assist devices, pacemakers, heart transplants and medications are available to treat heart failure patients, but outcomes for patients with heart failure are still worse than outcomes for the vast majority of cancer patients," says Rubinstein. "That's what we want to effectively change."
Other researchers in the UC College of Medicine also assisting the study are Mark Gilbert, MD, Mohit Kumar, James McNamara, PhD, Patrick Daly, MD, Sheryl Koch, PhD, Ginger Conway, Mohamed Effat, MD, and Jessica Woo.
The study performed at UC Medical Center is related to Rubinstein's work with probenecid to treat children and young adults with hypoplastic left heart syndrome. He received a $154,000 grant in June 2017 from the American Heart Association and Children's Heart Foundation to tackle this project with researchers at Cincinnati Children's. Hypoplastic left heart syndrome is a birth defect that affects normal blood flow through the heart.
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Monday, March 12, 2018

Gluten-free diet may help protect against neuropathic pain

A strict gluten-free diet may help protect against the nerve pain that some people with gluten sensitivity experience, according to a preliminary study released today that will be presented at the American Academy of Neurology's 70th Annual Meeting in Los Angeles, April 21 to 27, 2018.
"These findings are exciting because it might mean that a relatively simple change in diet could help alleviate painful symptoms tied to gluten neuropathy," said lead author Panagiotis Zis, MD, PhD, of the University of Sheffield in Sheffield, United Kingdom, and a member of the American Academy of Neurology. "While our study shows an association between a self-reported gluten-free diet and less pain, it does not show that one causes the other."
Gluten sensitivity has been associated with peripheral neuropathy -; a condition in which a person's peripheral nerves become damaged, often causing weakness, numbness and pain, typically in the hands and feet. When a person has nerve pain that can't otherwise be explained, and has a sensitivity to gluten, the diagnosis might be gluten neuropathy.
The study involved 60 people with an average age of 70 who had gluten neuropathy. They were asked about the intensity of their pain, their other neuropathy symptoms, their mental health and whether they followed a strict gluten-free diet. A total of 33 of the participants had pain with their neuropathy, or 55 percent.
People who were following a gluten-free diet were more likely to be free of pain than people who did not follow a strict gluten-free diet. A total of 56 percent of those without pain were on a gluten-free diet, compared to 21 percent of those with pain. After adjusting for age, sex and mental health status, researchers found that people following the strict diet were 89 percent less likely to have pain with their neuropathy than people not following the diet.
The study also found that people with painful gluten neuropathy scored significantly worse on the mental health assessment, which has a range of zero to 100 with 100 being best. Those with painful gluten neuropathy had an average score of 76, as opposed to the average score of 87 for those with painless gluten neuropathy.
"This study is promising because it shows that a gluten-free diet may help lower the risk of pain for people with gluten neuropathy," Zis said. "More research is needed to confirm these results and to determine whether the gluten-free diet led to the reduction in pain."


Friday, March 9, 2018

FDA Approves New Indication for Gilotrif (afatinib) in EGFR Mutation-Positive NSCLC

In continuation of my update on afatinib...

Boehringer Ingelheim  announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for Gilotrif (afatinib) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test. The new label includes data on three additional EGFR mutations: L861Q, G719X and S768I. The FDA granted Priority Review status to Gilotrif in evaluating this application.

Gilotrif, an oral, once-daily tablet, was previously approved in the U.S. for the first-line treatment of patients with NSCLC whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. In addition, Gilotrif is approved in the U.S. for patients with squamous cell carcinoma of the lung whose disease has progressed after treatment with platinum-based chemotherapy.
“With this expanded indication for Gilotrif, NSCLC patients whose tumors have certain EGFR mutations now have an approved therapy that specifically targets these mutations,” said Sabine Luik, M.D., senior vice president of Medicine & Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “This approval is a result of our company’s commitment to delivering meaningful treatment advances in areas with high unmet medical need and reflects the tireless efforts of physicians, researchers and patients who participated in our studies.”
The sNDA approval is based on a pooled analysis of three studies from the LUX-Lung clinical trial program (Phase II LUX-Lung 2 study and Phase III studies LUX-Lung 3 and LUX-Lung 6) that examined Gilotrif in NSCLC patients whose tumors have EGFR mutations, including L861Q, G719X or S768I. This analysis showed that Gilotrif was active in these EGFR mutations based on objective response rate, duration of response, disease control, progression-free survival and overall survival.
“Compared with other EGFR mutations, L861Q, G719X or S768I substitution mutations are associated with a poorer prognosis and limited treatment options,” said Edward Kim, M.D., Levine Cancer Institute, Carolinas HealthCare System. “The approval of Gilotrif as a targeted therapy for these additional non-resistant EGFR mutations significantly alters the treatment strategy for this population.”
To determine if a patient is eligible for Gilotrif, physicians must conduct a test for genetic mutations – also known as biomarker testing – to determine the type of EGFR mutation present.
“This approval is more welcome news for our lung cancer community,” said Laurie Fenton Ambrose, president and CEO of Lung Cancer Alliance. “These types of advances are helping expand access to treatment options for patients who might benefit from targeted therapies to fight their specific type of lung cancer.”

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FDA Approves New Indication for Gilotrif (afatinib) in EGFR Mutation-Positive NSCLC