Five months after getting a complete response letter from the U.S. FDA, Regeneron Pharmaceuticals Inc. has landed conditional European marketing approval for odronextamab, a bispecific antibody for treating lymphoma. Now named Ordspono, the approval is for treating adult patients with relapsed or refractory follicular lymphoma or diffuse large B-cell lymphoma, after two or more lines of therapy. The European Commission also formalized approval of Merck & Co. Inc.’s Winrevair (sotatercept) for the treatment of pulmonary arterial hypertension, making it the first activin signaling inhibitor to be approved in this indication. And ARS Pharmaceuticals Inc. gained clearance for its inhaled formulation of epinephrine, Eurneffy, as the first needle-free treatment for anaphylaxis.

FDA approves Vericel’s MACI for knee cartilage defects

Vericel Corp.’s MACI for repairing cartilage defects in the knee has been approved by the U.S. FDA via a supplemental biologics license application. Using a patient’s own cells cultured on a porcine collagen membrane, MACI Arthro is delivered arthroscopically and allows for repair of knee cartilage defects up to 4 cm².

Good Tryp: Psilocybin trial brings pain relief for fibromyalgia

All patients dosed in a phase IIa trial with Tryptamine Therapeutics Ltd.’s (Tryp) oral psilocybin (TRP-8802) trial reported an improvement in fibromyalgia pain severity, sleep, and pain interference. Patients reported other improvements in quality-of-life measures, such as sleep, physical activity and the ability to participate in daily social activities, Melbourne, Australia-headquartered Tryp said. Four out of five patients also reported a clinically meaningful reduction in anxiety and cognitive abilities. Conducted at the University of Michigan, the phase IIa open-label trial was conducted alongside psychotherapy.

Autoimmune disease latest focus of CAR T prospects

CAR T therapies for autoimmune disease has become an increasingly hot space. An up-and-comer that has gained Wall Street attention lately is Cartesian Therapeutics Inc. with Descartes-08, an autologous anti-BCMA mRNA candidate. But a league of other developers are busy in the zone as well.

Infectious disease index: Summit, Novavax, Inovio post gains

The BioWorld Infectious Disease Index (BIDI) has rebounded significantly from its lowest point this year, improving from a 25.12% drop in late April to a 7.66% decline by the end of July. Despite the recovery, BIDI still lagged behind the Nasdaq Biotechnology Index, which rose 10.82% by July's close, and the Dow Jones Industrial Average, which gained 8.25% year-to-date. In 2021, BIDI ended the year up 10.38%, then fell 83.57% in 2022 and dropped a further 43.48% in 2023.

Two enzymatic modulators could alleviate Gaucher disease

People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.

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