Monday, August 16, 2021

FDA Approves Twyneo (tretinoin and benzoyl peroxide) for the Treatment of Acne Vulgaris

In continuation  of my update on tretinoin  Sol-Gel Technologies, Ltd.   announced that the Food and Drug Administration (FDA) has approved its first proprietary drug product, Twyneo (tretinoin/benzoyl peroxide) cream, 0.1%/3%, indicated for the treatment of acne vulgaris in adults and pediatric patients nine years of age and older. Twyneo uses Sol-Gel’s patented technology to entrap tretinoin, a retinoid, and benzoyl peroxide within silica-based microcapsules to stabilize tretinoin from being degraded by benzoyl peroxide and to slowly release each of the active drug ingredients over time to provide a favorable efficacy and safety profile. Twyneo is patent protected until 2038. Sol-Gel has partnered with Galderma to commercialize Twyneo in the U.S. Sol-Gel expects to receive a regulatory milestone payment in conjunction with this approval and retains the option to regain U.S. commercialization rights five years following first commercialization in the U.S.

“The FDA approval of Twyneo underscores our ability to deliver innovative, proprietary drugs to the market,” stated Dr. Alon Seri-Levy, Co-Founder and Chief Executive Officer of Sol-Gel. “Based on the clinical data observed, we believe that Twyneo has the potential to change the treatment landscape for the tens of millions of patients suffering from acne vulgaris. With market leader, Galderma, handling the product launch of Twyneo, we are excited that Twyneo will soon be available to patients in the U.S.,” continued Dr. Seri-Levy. “We remain focused on obtaining FDA approval of Epsolay® (benzoyl peroxide), our other Galderma-partnered product – the approval of which has been delayed due to FDA’s COVID-19-related restrictions. We are also making progress on our innovative earlier stage programs for erlotinib, roflumilast and tapinarof with the intent of advancing them into the clinic,” he concluded.

“Galderma was founded forty years ago around a commitment to serve the dermatological needs of healthcare professionals and their patients,” said Baldo Scassellati Sforzolini, Global Head of Research & Development at Galderma. “Our heritage in acne dates back to our founding and we are excited to partner with Sol-Gel to bring yet another acne innovation to market for a condition that impacts up to 50 million Americans annually.1

“Twyneo combines, for the first time, two of the most commonly used topical agents available for the treatment of acne into a single application. Due to stability issues, these products don’t play well together, and we were never able to recommend even consecutive co-application of the two agents. Sol-Gel’s technology has solved this problem,” said Hilary Baldwin, M.D., Clinical Associate Professor of Dermatology, Rutgers Robert Wood Johnson School of Medicine, Medical Director, The Acne Treatment and Research Center and Past President of the American Acne and Rosacea Society. “The approval of Twyneo offers patients efficacy with these two products in a single convenient application. I believe physicians will look forward to adding Twyneo to their acne treatments toolbox.”

The New Drug Application (NDA) for Twyneo was approved by the FDA on July 26, 2021. The NDA was supported by positive results from two Phase 3, randomized, double-blind, vehicle-controlled, multi-center studies (NCT03761784, and NCT03761810), in which Twyneo demonstrated efficacy and a favorable tolerability profile in subjects nine years of age and older with facial acne vulgaris. Twyneo is the first FDA-approved fixed-dose combination of tretinoin and benzoyl peroxide. For more information, visit

FDA Approves Twyneo (tretinoin and benzoyl peroxide) for the Treatment of Acne Vulgaris

Friday, August 13, 2021

Spectrum Pharmaceuticals Receives Complete Response Letter from FDA for Rolontis (eflapegrastim)

Spectrum Pharmaceuticals, a biopharmaceutical company focused on novel and targeted oncology therapies, today announced receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the company’s Biologics License Application (BLA) for Rolontis® (eflapegrastim). The CRL cited deficiencies related to manufacturing and indicated that a reinspection will be necessary. The company is seeking further clarification from the FDA and plans to meet with the agency as soon as possible.

“We are disappointed with this outcome and look forward to fully understanding the remediation timelines for the program,” said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. “We continue to believe in Rolontis and plan to diligently complete the regulatory process to bring Rolontis to market.”

About Rolontis

Rolontis is a novel, long-acting granulocyte colony-stimulating factor (G-CSF) seeking an indication for the treatment of neutropenia in patients receiving myelosuppressive anti-cancer drugs. The BLA for Rolontis is supported by data from two identically designed Phase 3 clinical trials, ADVANCE and RECOVER, which evaluated the safety and efficacy of Rolontis in 643 early-stage breast cancer patients for the treatment of neutropenia due to myelosuppressive chemotherapy. In both studies, Rolontis demonstrated the pre-specified hypothesis of non-inferiority (NI) in duration of severe neutropenia (DSN) and a similar safety profile to pegfilgrastim. Rolontis also demonstrated non-inferiority to pegfilgrastim in the DSN across all 4 cycles (all NI p<0.0001) in both trials.

Friday, August 6, 2021

FDA Approves Lumakras (sotorasib) for KRAS G12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Amgen (NASDAQ: AMGN)  announced that the U.S. Food and Drug Administration (FDA) has approved Lumakras (sotorasib) for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. Lumakras has received accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

The FDA approval of Lumakras is a breakthrough moment for patients with KRAS G12C-mutated non-small cell lung cancer because there is now a targeted therapy for this common, but previously elusive, mutation," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "KRAS has challenged cancer researchers for more than 40 years with many deeming it as 'undruggable.' The Lumakras development program was a race against cancer for Amgen's scientists and clinical trial investigators who together have now successfully delivered this new medicine to patients in less than three years—from first patient dosed to U.S. regulatory approval."

e FDA approval of Lumakras is based on results from a subset of patients in CodeBreaK 100, the largest clinical trial conducted to date exclusively for patients with the KRAS G12C mutation. The trial demonstrated favorable efficacy and tolerability in 124 patients with KRAS G12C mutation-positive NSCLC who had disease progression after receiving an immunotherapy and/or chemotherapy. In the trial, 960 mg of Lumakras administered orally once-daily demonstrated an ORR (a proportion of patients with ≥ 30% decrease in tumor) of 36% (95% CI: 28-45) with 81% (95% CI: 73-87) of patients achieving disease control (percentage of patients who have achieved complete response, partial response and stable disease for more than three months). The median DoR was 10 months. The most common adverse reactions (≥ 20%) were diarrhea, musculoskeletal pain, nausea, fatigue, hepatotoxicity and cough. Adverse reactions resulting in permanent discontinuation of Lumakras occurred in 9% of patients.

"Sotorasib represents a major advancement in oncology and changes the treatment paradigm for patients with KRAS G12C-mutated non-small cell lung cancer," said Bob T. Li, M.D., Ph.D., MPH, principal investigator at Memorial Sloan Kettering Cancer Center. "Patients with non-small cell lung cancer who have progressed beyond first-line treatment face a poor prognosis and have limited treatment options available to them. Sotorasib delivers a new option for these patients, and it is the first KRAS-targeted therapy to be approved after nearly four decades of research."

NSCLC accounts for approximately 84% of the 2.2 million new lung cancer diagnoses each year worldwide, including approximately 236,000 new cases in the U.S.2,3 KRAS G12C is one of the most prevalent driver mutations in NSCLC, with about 13% of patients with non-squamous NSCLC in the U.S. having the KRAS G12C mutation.1

Amgen's Commitment to Comprehensive Biomarker Testing and Patient Support
About half of all patients with NSCLC harbor a targetable driver mutation, yet despite the integral role that biomarkers play in identifying patients who may benefit from targeted therapies, many patients are not tested.4,5

Amgen has partnered with two companies—Guardant Health and QIAGEN—to develop blood- and tissue-based companion diagnostics (CDx), respectively, for Lumakras. With the addition of these tests, patients and clinicians will have more options and flexibility for conducting KRAS G12C biomarker testing.

"Biomarker testing for patients with non-small cell lung cancer is critical because it informs a patient's treatment path with a personalized and tailored approach. The only way to identify the KRAS G12C mutation is to test for it, so I urge patients to ask their care teams about comprehensive biomarker testing. It is important that patients and their healthcare providers know that KRAS G12C is now an actionable mutation," said Andrea Ferris, president and CEO of LUNGevity. "Today's FDA approval of a therapy targeted for KRAS G12C, one of the most prevalent biomarkers in non-small cell lung cancer, brings hope to the many patients who carry this mutation and is a significant moment for the lung cancer community who need more innovative treatment options."

Thursday, August 5, 2021

Coffee Won't Upset Your Heartbeat. It Might Even Calm It

For decades, doctors have warned folks suffering from heart rhythm problems to avoid coffee, out of concern that a caffeine jolt might prompt a herky-jerky heartbeat.

But a large new study has found that most people can enjoy their morning joe or afternoon diet cola free from worry -- caffeine doesn't seem to increase most people's risk of arrhythmias.

"We see no evidence for this broad-based recommendation to avoid coffee or caffeine," said study co-author Dr. Gregory Marcus, associate chief of cardiology for research at the University of California, San Francisco. "There could be some individuals where caffeine is their trigger, but I think the growing evidence is those cases are actually quite rare."

In fact, results indicate that every additional cup of coffee a person drinks daily might lower their risk of arrhythmia by about 3% on average, according to the study published July 19 in JAMA Internal Medicine.

"The majority of people, even those with arrhythmias, should be able to enjoy their cup of coffee, and maybe there are some people for whom caffeine or coffee may actually help reduce their risk," Marcus said.

Coffee is one of the most widely consumed beverages worldwide, but its properties as a stimulant have prompted many doctors to warn heart patients against drinking java, Marcus said.

To see whether caffeine really can cause the heart to race or beat abnormally, Marcus and his colleagues analyzed data from more than 386,000 people participating in a long-term British health study.

Of that large group, about 17,000 developed a heart rhythm problem during an average follow-up of 4.5 years, researchers said.

All participants were asked about their coffee consumption when they entered the study. Researchers compared their response to their likelihood of developing an abnormal heart rhythm down the line.

The result: There was no link at all between caffeine and heart rhythm disturbances, even when researchers took into account genetic factors that might influence the way individuals metabolize caffeine.

"We could find no evidence on a population level that those who consumed more coffee or those exposed to more caffeine experienced a heightened risk for arrhythmias," Marcus said.

The study results show "there is absolutely some unsubstantiated dogma that coffee can cause arrhythmias," said Dr. Zachary Goldberger, an associate professor of cardiovascular medicine at the University of Wisconsin-Madison.

However, Goldberger cautioned against reading too much into what the study observed about caffeine's potential protective benefits, given that the effect was so small.

"I think the bottom line, based on these findings, is that coffee may not cause arrhythmias, but it doesn't necessarily protect against them either," said Goldberger, co-author of a commentary accompanying the study.

Further research is needed to suss out exactly how coffee affects the heart, and why it might protect against arrhythmias, Marcus said.

Coffee has anti-inflammatory effects, and it's well-known that inflammation can contribute to heart rhythm problems, Marcus said. It also might be that caffeine motivates some people to be more physically active, which reduces the risk of arrhythmia.

"We are probably not fully aware of the various mechanisms that may be relevant" to the relationship between caffeine and heart health, Marcus said.

Marcus said that he encourages his own heart rhythm patients to experiment with coffee.

"In many cases anecdotally, it doesn't make a difference," Marcus said. "For most, I have not found that it's an important trigger. They are very happy to receive this good news, especially those who enjoy coffee."

Both Marcus and Goldberger acknowledge there are probably some individuals who don't respond well to coffee, and their concerns should continue to be taken seriously.

"If a patient comes to clinic with palpitations, or symptoms of an arrhythmia, and asks whether caffeine or coffee plays a role, that is a personalized discussion," Goldberger said. "If a patient reports having palpitations that seem correlated with coffee or caffeinated beverages, these data don't give us the license to tell them not to try to limit coffee. But I think we could tell our patient that coffee doesn't place people at higher risk of heart rhythm disturbances

Coffee Won't Upset Your Heartbeat. It Might Even Calm It 

Wednesday, August 4, 2021

FDA Approves Soaanz (torsemide) for Edema Treatment in Patients with Heart Failure and Renal Disease

Sarfez Pharmaceuticals announced today approval by the U.S. Food and Drug Administration (FDA) of the Virginia-based company’s New Drug Application for Soaanz, a once-a-day improved formulation of the loop diuretic torsemide. The approved drug provides a new treatment option for patients suffering from heart failure who experience persistent edema, swelling in the lower limbs and/or abdomen, despite a loop diuretic therapy. Soaanz provides a longer duration of peak effects without causing excessive urination.

“Soaanz is specifically formulated to reduce excessive urination in heart failure patients. We believe that for many of these patients who are aged 65 and older, the new treatment option now approved by the FDA offers tremendous benefits,” said Salim Shah, PhD, JD, Founder and Chairman, Sarfez Pharmaceuticals. “We look forward to launching Soaanz in the next few months, bringing an effective new treatment option to heart failure patients.”

Soaanz offers an alternative treatment for heart failure patients who skip loop diuretic treatment due to concerns with excessive urination, as well as patients with chronic kidney disease. Loop diuretics are the drug of choice for the treatment of volume overload caused by heart failure and chronic kidney disease. Soaanz is an oral once-a-day tablet with an extended duration of peak effect and with a longer time to reach the peak effect.

“Sarfez Pharmaceuticals benefited from grant funding made available by the Small Business Administration and National Institute of Diabetes and Digestive and Kidney Diseases during our development of Soaanz,” said Dr. Shah. “We are thankful for that support, and we plan to transfer that benefit on to our patients by offering Soaanz at a reasonable price. This was an important decision for our team to make because most heart failure patients are insured under Medicare programs and have limited disposable income. We feel that lowering our costs to help improve care for these patients is our obligation as a healthcare company.”

For more information on Soaanz, please visit:

Tuesday, August 3, 2021

FDA Approves Rezipres (ephedrine hydrochloride injection) for Hypotension During Anesthesia

Eton Pharmaceuticals, Inc (Nasdaq: ETON) today announced that the U.S. Food and Drug Administration (FDA) has approved Rezipres (ephedrine hydrochloride injection) for the treatment of clinically important hypotension occurring in the setting of anesthesia.

“We are excited to see the approval of Rezipres, which is now our second FDA-approved ready-to-use hospital injectable product. We believe ready-to-use injectable products provide a compelling benefit to hospitals and reduce the need for hospitals to rely on unapproved compounded products,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “This innovative sulfite-free formulation has been successfully sold in Europe for years, and we are excited to make it available to U.S. patients shortly.”

About Eton Pharmaceuticals

Eton Pharmaceuticals, Inc. is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The company currently owns or receives royalties from four FDA-approved products, including ALKINDI® SPRINKLE, Biorphen®, Rezipres®, and Alaway® Preservative Free, and has five additional products that have been submitted to the FDA.

Monday, August 2, 2021

FDA Approves Astepro Allergy (azelastine) Nasal Spray for Over-the-Counter Use

In continuation of my update on Azelastine

Bayer announced today that the U.S. Food and Drug Administration (FDA) has approved Astepro Allergy (Azelastine HCI .15%) as an over-the-counter (OTC) product for the temporary relief of nasal congestion, runny nose, sneezing and itchy nose due to hay fever or other upper respiratory allergies. With the FDA’s approval, Astepro Allergy becomes the first and only steroid free, antihistamine nasal spray for allergies available OTC in the United States for adults and children 6 years of age and older.

Astepro® is currently only available with a prescription in the U.S. Today’s OTC approval of Astepro® Allergy will provide more than 50 million Americans that suffer from allergies each year over-the-counter access to this steroid free nasal spray optioni. Currently, OTC allergy medications includes three major classes: antihistamines, intranasal steroids and mast cell stabilizers. Astepro® Allergy will be the first and only OTC antihistamine nasal spray for indoor and outdoor allergy relief upon the OTC switch.

“At Bayer, we help make self-care a reality for billions through everyday healthcare. This new differentiated product is one step in that direction, with the potential to help millions of people in the U.S. that suffer from seasonal allergic rhinitis,” said Catherine Vennat, VP/GM Bayer Consumer Health U.S. “With Astepro® Allergy, allergy sufferers can get relief of allergy nasal symptoms in an over-the-counter treatment option without steroids or decongestants that will last up to 24-hours. It will bring a crucial differentiated allergy product to the Bayer allergy portfolio to address unmet consumer needs with allergic rhinitis.”

This OTC formulation is full prescription strength and includes a number of benefits including flexible once or twice daily dosing that provides up to 24-hour relief of nasal congestion, running nose, sneezing and itchy nose from indoor and outdoor allergies.