FDA Action Alert: Amylyx's Moment of Truth and Dupixent Seeks another Nod

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The FDA might only have two PDUFA dates on the calendar this week, but all eyes in the neurodegenerative disease community will be on the first one: Amylyx's AMX0035 for ALS. 

September 29: Amylyx 

Following not one, but two advisory committee votes, the FDA will consider the New Drug Application for Amylyx Pharmaceuticals' AMX0035 for amyotrophic lateral sclerosis (ALS).

In March, the drug went before the FDA’s Central Peripheral and Central Nervous System Drugs Advisory Committee, which voted 6 to 4 against its recommendation, based mostly on the clinical trial design.

In June, the FDA extended the review timeline to take into consideration additional data.

On September 8, the same committee reconvened to assess post hoc analysis of data that demonstrated a 10.6-month longer median survival duration for patients on active treatment. The committee voted 7-2 in support of the drug.

Their decision was largely based on new data analysis from the Phase II CENTAUR trial, the dire nature of the disease and the significant unmet medical need. There is also an ongoing Phase III PHOENIX trial, which could potentially confirm the data. 

“The new survival analyses presented today support the robustness of the overall survival result, as the benefit is consistently observed across multiple analyses," said Josh Cohen and Justin Klee, co-CEOs of Amylyx in a statement provided to BioSpace

September 30: Sanofi and Regeneron

Sanofi and Regeneron have a target action date of September 30 for their supplemental Biologics License Application (sBLA) for Dupixent (dupilumab) as a treatment for adults with prurigo nodularis. The chronic inflammatory skin disorder is marked by extreme itching and skin lesions.

The sBLA is built on data from two pivotal Phase III trials in adults with uncontrolled prurigo nodularis (PRIME2 and PRIME). Both studies hit the primary and key secondary endpoints, with the drug significantly demonstrating improved signs of disease and symptoms compared to placebo. The safety profile was consistent with that of the drug in atopic dermatitis. The most common adverse event was conjunctivitis.

Dupixent is a fully human monoclonal antibody against the interleukin-4 and 13 pathways. It is indicated for a number of inflammatory disorders, including asthma, atopic dermatitis, chronic rhinosinusitis with nasal polyposis and eosinophilic esophagitis.

Gil Yosipovitch, M.D., professor of dermatology, Miller School of Medicine, University of Miami, director of the Miami Itch Center and principal investigator of the Phase III PRIME trial, commented on the data. 

"These positive results...in prurigo nodularis confirm inhibiting IL-4 and IL-13 can significantly reduce the unrelenting itch and extensive severe skin lesions that often impair patient quality of life," he said in a September 8 statement.

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