FDA Review: Amylyx, Sarepta, Sanofi-Regeneron and More

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, Regeneron GE Healthcare and more. 

Here’s a look.

Sept. 30

Immuneering Corporation’s IND for IMM-1-104 was given the green light for a Phase I/IIa trial for advanced RAS mutant solid tumors.

Sept. 29

Amylyx’s combination of sodium phenylbutyrate and taurusodiol was approved to treat amyotrophic lateral sclerosis (ALS). It will be marketed under the brand name Relyvrio.

Sarepta Therapeutics submitted a BLA to the FDA for accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy (DMD). SRP-9001 is a gene therapy being developed in partnership with Roche. The BLA was based on the expression of SRP-9001 dystrophin protein as a surrogate endpoint that would probably predict clinical benefit. 

Napo Pharmaceuticals announced the FDA greenlit its Phase I trial of NP-300 for treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy.

GE Healthcare received FDA 510(K) clearance of its AIR Recon DL for 3D and PROPELLER imaging sequences, which are used by almost all MRI clinical procedures.

InflammX Therapeutics submitted an IND Amendment to launch clinical trials with Xiflam, an oral NLRP3 inflammasome inhibitor with broad applications, but initial focus on Diabetic Macular Edema (DME), the intermediate stage of Age-related Macular Degeneration (iAMD) and the Geographic Atrophy form of AMD.

InflaRx submitted an Emergency Use Authorization request for vilobelimab for the treatment of critically ill COVID-19 patients.

C4 Therapeutics received the go-ahead to launch a Phase I/II trial for CFT1946 to treat BRAF-V600 mutant solid tumors.

Alzamend Neuro submitted an IND application to the FDA for ALZN002, an immunotherapy to treat mild to moderate dementia of the Alzheimer’s type.

Sept. 28

Sanofi and Regeneron’s Dupixent (dupilumab) was approved for adults with prurigo nodularis.

Citius Pharmaceuticals submitted a BLA for denileukin diftitox, an engineered IL-2-diphtheria toxin fusion protein for persistent or recurrent cutaneous T-cell lymphoma.

Celltrion USA’s Vegzelma (bevacizumab-adcd), a biosimilar to Avastin, was approved by the FDA for treatment of six types of cancer: metastatic colorectal cancer; recurrent or metastatic non-squamous non-small cell lung cancer; recurrent glioblastoma; metastatic renal cell carcinoma; persistent, recurrent or metastatic cervical cancer; and epithelial ovarian, fallopian tube, or primary peritoneal cancer.

Pharming Group announced the FDA accepted for priority review its NDA for leniolisib to treat activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years and older.

Mallinckrodt Pharmaceuticals submitted a 510(K) premarket notification application for an investigational inhaled nitric oxide delivery system for inhalation of INOmax (nitric oxide) gas.

OxyNov filed a 510(k) submission with the FDA for the FreeO2 device, an oxygen therapy device.

Sept. 27

Vertex Pharmaceuticals and CRISPR Therapeutics concluded discussions with the FDA, and the FDA granted exagamglogene autotemcel a rolling review for the potential treatment of sickle cell disease and transfusion-dependent beta thalassemia.

Blue Earth Diagnostics announced the FDA accepted its NDA application for 19F-rhPSMA-7.3, a radiohybrid Prostate-Specific Membrane Antigen-targeted PET imaging agent.

Bellerophon Therapeutics reported the FDA accepted its proposal to reduce the study size of its ongoing REBUILD Phase III trial of INOpulase for fibrotic Interstitial Lung Disease. The new study size will be 140 patients.

Ayala Pharmaceuticals was granted Fast Track designation for AL102 for treatment of progressing desmoid tumors.

Sept. 26

Atea Pharmaceuticals announced the FDA granted Fast Track Designation to AT-752, an oral therapy for dengue virus infection.

Pfizer and BioNTech reported they had completed an Emergency Use Authorization submission to the FDA for a 10-microgram dose of its Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine for kids 5 through 11 years of age.

CatalYm received the greenlight to expand its ongoing Phase II clinical program to include U.S. sites. The ongoing GDFATHER-2 program is studying visugromab in combination with an anti-PD1 antibody in advanced solid tumors that are relapsed/refractory to previous anti-PD1/PD-L1 treatment.

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