FDA Updates for the Week of July 4, 2022

FDA expands label for Krystexxa.

The FDA has approved the supplemental biologics license application (sBLA) expanding the labeling to include Krystexxa (pegloticase) injection co-administered with methotrexate. Developed by Horizon Therapeutics, Krystexxa is a biologic indicated to treat adult patients with uncontrolled chronic gout.

The approval for use with methotrexate is based on the results from the MIRROR trial, which found that 71% of patients randomized to receive Krystexxa with methotrexate versus 39% of patients who received Krystexxa with placebo achieved the primary endpoint. The endpoint measured was the proportion of serum uric acid responders during month six. Additionally, 60% of patients who received the combination achieved a complete response during month 12 compared with 31% of patients who received the therapy with placebo.

FDA grants priority review for lecanemab for Alzheimer’s disease.

The FDA has accepted the biologics license application (BLA) under the accelerated approval pathway for lecanemab to treat patients with Alzheimer’s disease. Eisai's application, which was completed in early May 2022, has been granted priority peview, with a Prescription Drug User Fee Act (PDUFA) action date of Jan. 6, 2023.

Lecanemab is an investigational anti-amyloid beta antibody being developed jointly by Eisai and Biogen for the treatment of mild cognitive impairment due to Alzheimer’s disease. It is the second Alzheimer's therapy to come from the collaboration behind Aduhelm.

The submission is based on clinical, biomarker and safety data from the proof-of-concept phase 2b in 856 people with early Alzheimer’s disease with confirmed presence of amyloid pathology, biomarker and safety data from an open-label extension study, and blinded safety data from the confirmatory phase 3 study.

FDA grants priority review for novel immunotherapy for follicular lymphoma.

The FDA has accepted the Genentech’s biologics license application and granted priority review for mosunetuzumab for the treatment of adults with relapsed or refractory follicular lymphoma who have received at least two prior systemic therapies. The FDA is expected to make a decision by Dec. 29, 2022.

Follicular lymphoma is the most common slow growing form of non-Hodgkin’s lymphoma, a type of blood cancer. Mosunetuzumab is a potential first-in-class bispecific antibody. It is designed to target CD20 on the surface of B cells and CD3 on the surface of T cells. This redirects a patient’s existing T cells to release cytotoxic proteins into the B cells.

The European Commission granted conditional marketing authorization in June 2022 for mosunetuzumab, marketed as Lunsumio, for this indication.

The submission is based on positive results from the pivotal phase ½ GO29781 study of mosunetuzumab, which showed high complete response rates, with the majority of responders (57%) maintaining responses for at least 18 months, and manageable tolerability in people with heavily pretreated follicular lymphoma.

FDA convenes advisory committee for the second time for ALS therapy.

The FDA plans reconvene the Peripheral and Central Nervous System Drugs Advisory Committee to discuss Amylyx Pharmaceuticals’ new drug application for AMX0035 for the treatment of patients with amyotrophic lateral sclerosis (ALS). The committee meeting, scheduled for Wednesday, Sept. 7, 2022, will discuss additional analyses of data from clinical studies.

This is the second meeting of the advisory committee, which will discuss new analysis that the company announced in May 2022 of long-term survival analysis of the phase 2 CENTAUR trial. This new analysis used a different model, the rank-preserving structural failure time model, a method frequently used in oncology to account for placebo crossover. Using this model, AMX0035 is estimated to provide a 10.6-month longer median survival duration for participants.

The FDA had extended the review timeline to Sept. 29, 2022 (formerly June 29, 2022), to allow additional time review this additional analysis.

Bausch + Lomb submits NDA for dry eye disease therapy.

Bausch + Lomb and Novaliq have submitted a new drug application (NDA) to the FDA seeking approval for NOV03 (perfluorohexyloctane), an investigational treatment for patients with dry eye disease (DED) associated with Meibomian gland dysfunction (MGD).

Perfluorohexyloctane 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. If approved, it will be the first to address signs and symptoms of dry eye disease.