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FDA Updates for Week of Sept. 5, 2022


In COVID-19 news, the FDA indicated Eiger’s treatment not supported by data. An advisory committee switched its vote to yes for new ALS drug. The FDA approved Stimufend, a biosimilar to Neulasta, as well as Imfinzi for biliary tract cancer, a longer-lasting treatment for frown lines, an oral suspension for gastric ulcers, and generics of Revlimid. The agency accepted applications for novel dry eye disease and alopecia therapies. Additionally, Seres completed rolling BLA for microbiome therapeutic.

FDA advisory committee supports approval of AMX0035 for ALS.

Despite additional data that some members said only added modestly to the evidence, an FDA advisory committee voted to support approval of Amylyx’ AMX0035 (sodium phenylbutyrate and taurursodiol). In a 7 to 2 vote, the Peripheral and Central Nervous System Drugs Advisory Committee late on Wed. Sept. 7, 2022, supported approval of Amylyx Pharmaceuticals’ therapy to treat patients with amyotrophic lateral sclerosis (ALS).

While not binding, the committee’s recommendation will be considered by the FDA in its review of the pending new drug application (NDA) for AMX0035.The Prescription Drug User Fee Act (PDUFA) target action date is Sept. 29, 2022.

This was the second time the advisory committee considered the question of whether the data support the effectiveness of AMX0035. At the first advisory committee meeting in March 2022 in which six of the 10 members voted no on the question of whether the open-label extension of the phase 2 CENTAUR trial established a conclusion that AMX0035 is effective in the treatment of patients with ALS.

After Wednesday’s vote, several committee members mentioned they were swayed in part by the additional analysis presented by Amylyx. Some indicated the unmet and the seriousness of ALS, as well as encouraging signals from the ongoing phase 3 PHOENIX trial, encouraged them to switch their no votes to yes.

AMX0035 is approved in Canada with the brand name Albrioza, and a marketing application is also pending in Europe.

FDA approves Stimufend, a biosimilar to Neulasta.

The FDA has approved Fresenius Kabi’s biosimilar Stimufend (pegfilgrastim-fpgk) for use in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. The company expects to launch the product as a prefilled syringe early next year. Stimufend is a biosimilar of Amgen’s Neulasta. The company is also developing on-body injector for Stimufend.

Stimufend is Fresenius Kabi’s first U.S.-approved therapy in its biosimilar portfolio.

Pegfilgrastim biosimilar is a supportive care medicine for patients with non-myeloid cancer. It stimulates the growth of certain white blood cells, which are essential to prevent or fight infections, a common life-threatening risk in patients receiving myelosuppressive chemotherapy.

The approval for Stimufend is based on a review of a comprehensive data package and a totality of evidence that demonstrated a high degree of similarity with the reference product.

FDA: Eiger’s COVID-19 treatment not supported by data.

The FDA is requiring additional data and information on Eiger BioPharmaceuticals’ request for emergency use authorization (EUA) of peginterferon lambda to treat patients with mild-to-moderate COVID-19. Following a pre-EUA information exchange with the FDA regarding the phase 3 TOGETHER study of peginterferon lambda for COVID-19, the agency has indicated that it is not yet able to determine whether the criteria for the submission of an application and issuance of an EUA are likely to be met.

The company has generated new data and analyses from the TOGETHER study that it plans to discuss with FDA, including further statistical modeling and efficacy analyses of the study’s primary and secondary endpoints in patients treated within three days of symptom onset. The endpoint of hospitalization due to COVID-19 and all-cause mortality for patients treated within three days of symptom onset is consistent with the endpoint used to authorize other therapeutics for emergency use.

The original efficacy analysis of the data generated from the TOGETHER study was based on dosing peginterferon lambda within seven days of symptom onset. Eiger also plans to provide the FDA new additional analyses of long-term follow-up data, including rates of rebound and incidence of long COVID, as well as an indirect comparative analysis of mortality and hospitalizations in vaccinated patients when treated with peginterferon lambda compared with other therapeutics authorized for emergency use.

Peginterferon lambda is a first-in-class, type III interferon that stimulates immune responses that are critical for the development of host protection during viral infections. Eiger licensed worldwide rights to lambda from Bristol Myers Squibb.

FDA approves Imfinzi for metastatic biliary tract cancer.

The FDA has approved AstraZeneca’s Imfinzi (durvalumab) to treat adult patients with locally advanced or metastatic biliary tract cancer in combination with chemotherapy (gemcitabine plus cisplatin). Imfinzi is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins.

Biliary tract cancer is a group of rare and aggressive cancers. About 23,000 people in the United States are diagnosed with these cancers each year. These patients have a poor prognosis, with about 5% to 15% of patients surviving five years.

The approval was based on the results from the TOPAZ-1 phase 3 trial. In an interim analysis, Imfinzi plus chemotherapy reduced the risk of death by 20% versus chemotherapy alone. An estimated one in four patients treated with Imfinzi plus chemotherapy were still alive at two years compared with one in 10 treated with chemotherapy alone.

In July 2022, Imfinzi plus chemotherapy was added to the NCCN Clinical Practice Guidelines in Oncology as a Category 1 preferred regimen as 1st-line therapy for locally advanced or metastatic biliary tract cancer based on the data from TOPAZ-1.

FDA approves oral suspension for gastric ulcers.

The FDA has approved Azurity Pharmaceuticals’ Konvomep (omeprazole and sodium bicarbonate). Konvomep is an oral suspension to treat active benign gastric ulcer and reduction of risk of upper gastrointestinal bleeding in critically ill patients. Azurity officials expect Konvomep will become commercially available in the first quarter of 2023.

In July 2022, the FDA approved Azurity’s Zonisade, an oral suspension of zonisamide to treat partial seizures in adults and pediatric patients aged 16 years and older with epilepsy. This is the first oral liquid form of zonisamide.

FDA approves longer-lasting treatment for frown lines.

The FDA has approved Daxxify (daxibotulinumtoxinA-lanm) for injection for the temporary improvement of moderate-to-severe frown lines (glabellar lines) in adults. Developed by Revance Therapeutics, Daxxify is an acetylcholine release inhibitor and neuromuscular blocking agent stabilized with the company’s Peptide Exchange Technology. It is free of both human serum albumin and animal-based components.

The approval was based on the data generated in the SAKURA phase 3 clinical trial program (SAKURA 1,2,3). The median duration of effort was six months. Some patients maintained treatment results at nine months, with results seen as early as one day after treatment, typically seen within two days.

FDA, EMA accept submissions for alopecia therapy.

The FDA has accepted for Pfizer’s new drug application (NDA) for ritlecitinib for adults and adolescents 12 years of age and older with alopecia areata. The FDA is expected to make a decision in the second-quarter 2023. Additionally, the European Medicines Agency (EMA) has also accepted the marketing authorization application (MAA) for ritlecitinib in the same patient population with a decision anticipated in the fourth-quarter 2023.

Alopecia areata is an autoimmune disease where the immune system attacks the body’s hair follicles, causing hair to fall out. Alopecia areata affects about 6.8 million people in the United States and around 147 million people globally.

Ritlecitinib is an investigational oral, once-daily treatment that is the first in a new class of oral highly selective kinase inhibitors that is a dual inhibitor of the TEC family of tyrosine kinases and of Janus kinase 3 (JAK3).

FDA accepts NDA for novel dry eye disease therapy.

The FDA has accepted Bausch + Lomb’s new drug application (NDA) for NOV03 (perfluorohexyloctane) associated with Meibomian gland dysfunction (MGD). The therapy is a first-in-class eye drop with a novel mechanism of action. It prevents excessive tear evaporation and has the ability to restores tear film balance. It stabilizes the lipid layer for hours to protect the tear film and has the ability to penetrate the Meibomian glands. NOV03 has been assigned a Prescription Drug User Fee Act (PDUFA) action date of June 28, 2023.

Bausch + Lomb submitted the NDA for NOV03 in July 2022 based on two pivotal phase 3 trials, (GOBI and MOJAVE), both of which demonstrated statistically significant improvement vs. control for both primary and key secondary sign and symptom endpoints.

Seres completes rolling BLA for microbiome therapeutic.

Seres Therapeutics has completed the rolling submission process for its biologics license application (BLA) to the FDA for SER-109 for the prevention of recurrent C. difficile infection. C. difficile is a bacterium that causes diarrhea and colitis. The CDC estimates that there are about half a million infections a year, and one in six patients will have a second infection within two to eight weeks. In those over the age of 65, one in 11 patients with C. difficile will die within one month.

SER-109 is an oral microbiome therapeutic consisting of a purified Firmicutes spores, which normally live in a healthy microbiome. SER-109 is designed to modulate the disrupted microbiome so that it resists C. difficile growth. The SER-109 manufacturing purification process is designed to remove unwanted microbes, reducing the risk of pathogen transmission

Dr. Reddy’s launches, Cipla receives approval for generic Revlimid.

Dr. Reddy’s Laboratories has launched lenalidomide capsules, a generic equivalent of Bristol Myers Squibb’s Revlimid (lenalidomide) capsules, which is used to treat patients with multiple myeloma, mantle cell lymphoma, and myelodysplastic syndromes, as a maintenance therapy following stem cell transplant.

Dr. Reddy’s received a first-to-market 180 days of exclusivity for the 2.5 mg and 20 mg strengths. Dr. Reddy’s lenalidomide is available in strengths of 2.5 mg, 5 mg, and 10 mg in a bottle-count size of 28, as well as 15 mg, 20 mg, and 25 mg strengths, each in a bottle-count size of 21.

Additionally, the FDA approved Cipla’s generic version of Revlimid capsules in 5 mg, 10 mg, 15 mg and 25 mg strengths. The company said the product will be shipping soon. Teva Pharmaceuticals became the first to launch a generic of Revlimid in March 2022 in 5 mg, 10 mg, 15 mg, and 25 mg strengths.

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