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FDA Roundup: This Weeks FDA Updates (August 15-21)

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FDA rejects hemophilia gene therapy
The U.S. Food and Drug Administration issued a Complete Response Letter to the BioMarin Pharmaceutical’s Biologics License Application for valoctocogene roxaparvovec gene therapy for severe hemophilia A, according to a press release.

Possibly approval is likely delayed till late in 2022.

“We remain committed to the hemophilia community and to leading the way to the first ever gene therapy in hemophilia A,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin in the press release.  “We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter.  We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A.”

Read the full press release here.

FDA approves treatment for neuromyelitis optica spectrum disorder
The U.S. Food and Drug Administration has approved satralizumab-mwge (Enspryng; Genentech) for adults with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disorder of the central nervous system that damages the optic nerve and spinal cord, according to a press release.

It is the only approved treatment for NMOSD that inhibits interleukin-6 receptor activity, using novel recycling antibody technology.

Aatralizumab-mwge is administered by subcutaneous injection every 4 weeks and can be given at home by patient or caregiver.

Approval is based on results from the phase 3 SAkuraStar and SAkuraSky clinical trials, where the effects of  satralizumab-mwge in adults with AQP4 antibody positive NMOSD were sustained for 96 weeks and significantly reduced the risk of relapse compared with placebo as a monotherapy and when used concurrently with baseline immunosuppressant therapy—a common way to manage NMOSD symptoms associated with relapses.

“Today’s FDA approval of Enspryng, the first subcutaneous NMOSD treatment using novel recycling antibody technology, builds upon the work we’ve done in multiple sclerosis with Ocrevus to develop first-in-class medicines and further the scientific understanding of neuroimmunological diseases,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development. “We thank the NMOSD community, including patients and investigators who participated in Enspryng clinical trials.”

View the full press release here.

FDA rejects filgotinib for moderate to severe active rheumatoid arthritis
The U.S. Food and Drug Administration has issued a complete response letter for the New Drug Application for filgotinib for moderately to severely active rheumatoid arthritis (RA), according to a press release from Gilead Sciences, Inc.

According to the press release, the FDA requested data from the MANTA and MANTA-RAy studies which assessed the impact filgotinib has on sperm parameters. In addition, the FDA also expressed concerns on the overall benefit/risk profile of the filgotinib 200 mg dose.

“We are disappointed in this outcome and will evaluate the points raised in the complete response letter for discussion with the FDA. We continue to believe in the benefit/risk profile of filgotinib in RA, which has been demonstrated in the FINCH Phase 3 clinical program,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences in a statement.

Read the full press release here.

FDA approves daratumumab-based combination treatment for relapsed/refractory multiple myeloma
The U.S. Food and Drug Administration has approved of daratumumab (Darzalex) in combination with carfilzomib (Kyprolis) and dexamethasone (DKd) for the treatment of adult patients with relapsed/refractory multiple myeloma who have received 1 to 3 previous lines of therapy, according to a press release by Janssen Pharmaceutical.

“The significant increase in progression-free survival (PFS) seen among patients receiving the DKd regimen supports the use of this new combination for patients with relapsed and refractory multiple myeloma. We continue to advance effective regimens for the most critical patients who have already relapsed,” said Saad Z. Usmani, MD, Division Chief of Plasma Cell Disorders, Atrium Health’s Levine Cancer Institute, and principal investigator of the CANDOR study, in the press release. “The DKd regimen fills an important gap in the treatment landscape, as many patients may relapse following an immunomodulatory drug-based therapy, such as lenalidomide-containing regimens, and therefore new therapeutic options are needed.”

Read the full press release here.