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Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.
Despite the recent emergence of a new drug class, schizophrenia remains one of the most challenging neuropsychiatric diseases to treat—and Cobenfy’s first steps into the market indicate there is still room to grow.
While there are several FDA-approved psychiatric medicines on the market, many patients still struggle to achieve lasting symptom relief. Psychedelics could help fill this gap.
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Read our takes on the biggest stories happening in the industry.
The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
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Vaderis is hoping that engasertib will become the first approved medicine for hereditary hemorrhagic telangiectasia, a rare condition that can cause spontaneous bleeding.
The national growth vehicle isn’t biotech‑dedicated, but channeling capital toward life sciences would amplify Austria’s strong research hubs, nondilutive funding landscape and reputation as a champion in science and technology.
Bristol Myers Squibb’s $2.3 billion Houston manufacturing campus is part of the company’s larger $40 billion investment designed to bolster American innovation and manufacturing.
A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.
Jazz Pharmaceuticals is reaching into its wallet once again, this time buying Actio Biosciences and an experimental therapy for KCNT1+ epilepsy, a form of the disease with no FDA-approved treatments.
Definium Therapeutics’ closely watched anxiety readout could further validate its single-dose LSD candidate, while Compass Pathways advances a psilocybin program through a rolling FDA filing.
News of the joint venture comes months after AstraZeneca vowed to spend $15 billion in China by the end of the decade.
Patients treated with Tenax Therapeutics’ heart failure drug TNX-103 did not show significantly better functional performance—though Leerink analysts found biomarker improvements and subgroup analyses in elderly and more severe patients “very encouraging.”
BlossomHill Therapeutics will use its $150 million IPO proceeds to power the Phase 1/2 development of a non-small cell lung cancer drug, while Latigo Therapeutics will bring in more than $345 million for its late-stage non-opioid pain pill.
BioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements.