The U.S. FDA’s Oncology Drugs Advisory Committee is deliberating over label expansions for two CAR T therapies: Carvykti (ciltacabtagene autoleucel) from Johnson & Johnson and the Bristol Myers Squibb Co. product Abecma (idecabtagene vicleucel). Both are approved as fifth-line treatments in relapsed/refractory multiple myeloma, and the companies want to get their respective therapies cleared for use earlier in the disease’s progression. Carvykti was given the go-ahead in February 2022, and Abecma in March 2021.

Madrigal’s Rezdiffra wins first FDA nod in NASH, liver fibrosis

Shares of Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) opened March 15 up 20% on news that the U.S. FDA approved its resmetirom as the first treatment for nonalcoholic steatohepatitis (NASH), an advanced form of nonalcoholic fatty liver disease that has been steadily increasing in global prevalence. Branded Rezdiffra, the liver-directed THR-beta agonist gained accelerated approval for use in conjunction with diet and exercise to treat adults with noncirrhotic NASH with moderate to advanced liver fibrosis, specifically stages F2 and F3. Madrigal expects Rezdiffra to be available to patients in April.

BMS and Mirum add approvals to established drugs

Two sNDAs have received U.S. FDA approval to further expand their treatment indications. Bristol Myers Squibb Co.’s Breyanzi (lisocabtagene maraleucel), a CD19-directed CAR T-cell therapy with a 4-1BB costimulatory domain, received accelerated approval from the FDA for treating adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma. The one-time infusion had a March 14 PDUFA date. Also, Mirum Pharmaceuticals Inc.’s Livmarli (maralixibat), an ileal bile acid transporter inhibitor, for treating progressive familial intrahepatic cholestasis, had a March 13 PDUFA date. Livmarli is also approved for treating cholestatic pruritus in patients with Alagille syndrome in the U.S., Europe, Canada and other regions across the globe.

Neurological stocks navigate turbulence despite strong 2023 performance

In 2023, the BioWorld Neurological Diseases Index (BNDI) closed with a 4.36% increase, outperforming the Nasdaq Biotechnology Index (up 3.74%) but falling short of the surge seen in the Dow Jones Industrial Average (up 13.7%). The rebound marked a significant turnaround from November, when BNDI recorded an 18.37% year-to-date decline.

FDA clears Beigene’s PD-1 inhibitor Tevimbra in esophageal cancer

The U.S. FDA has approved Beigene Co. Ltd.’s Tevimbra (tislelizumab-jsgr) as a monotherapy for treating adults with unresectable or metastatic esophageal squamous cell carcinoma following prior chemotherapy that did not include a PD-1 inhibitor. The approval marks the first U.S. approval for tislelizumab. It is the first drug candidate produced from Beigene’s immuno-oncology program and is being developed internationally as a monotherapy and in combination with other therapies to treat a broad array of both solid tumor and hematologic cancers. It is expected to be available in the U.S. in the second half of 2024, Beigene said.

Needle-free epinephrine prodrug yields strong phase III PK data

Aquestive Therapeutics Inc.’s oral epinephrine prodrug to treat life-threatening allergic reactions, Anaphylm, produced a faster time to maximum concentration than currently available auto-injectors in healthy adults, early pivotal phase III data show, meeting both pharmacokinetic primary and secondary endpoints. If approved, the sublingual film would be the first oral offering to treat severe allergies including anaphylaxis, providing patients with an alternative, easier-to-carry dosing option than epinephrine auto-injectors such as Viatris Inc.’s Epipen and Sanofi SA’s Auvi-Q, which come with limitations.

February biopharma M&As soar to $18B, outpacing 2023 average

In February, deals formed by biopharma firms reached to $7.76 billion, down from January, which marked the sixth-highest month in BioWorld’s records going back to 2016. Meanwhile, biopharma M&As amounted to $18.22 billion for the month, positioning it among the top 10 highest-value months in the past four years.

Also in the news

60 Degrees, Aprea, Atrogi, Cognition, Cybin, Dr. Vince Clinical Research, Eupraxia, First Wave, Gynica, Immuneering, Immunogenx, Keros, Mapp, Mirum, Newron, Nuvectis, Ocuterra, Ono, Partex, Poseida, Probiogen, Recce, Sanofi, Scisparc, Sellas, Sibylla, Spruce, Xcovery