Pharmaceutical Industry Advances: New Drug Approvals, Gene Therapy Initiatives, and Manufacturing Expansions

Crinetics' Palsonify Challenges Established Acromegaly Treatments

Crinetics Pharmaceuticals has received FDA approval for Palsonify (paltusotine), the first once-daily oral therapy for acromegaly. The drug is cleared for adult patients who are not eligible for corrective surgery or have not adequately responded to it. With a broad label and an annual list price of $290,000, Palsonify is poised to compete with established treatments from pharmaceutical giants like Novartis and Pfizer. Leerink Partners analyst Joseph Schwartz predicts peak annual sales of $375 million in the U.S. and Europe.

Government Initiatives Boost Gene Therapy Development

The FDA and the Advanced Research Projects Agency (ARPA) have announced new programs to accelerate gene therapy research and development. The FDA has published draft guidance outlining accelerated approval pathways, post-approval requirements, and new clinical trial designs for gene therapies. Simultaneously, ARPA has introduced two programs aimed at expediting development and improving manufacturing processes to reduce production costs for these complex medicines.

Pharmaceutical Companies Embrace Direct-to-Consumer Sales

Bristol Myers Squibb is set to offer its psoriasis drug Sotyktu online at a significantly discounted price starting January 2026. The initiative will provide uninsured, underinsured, or out-of-pocket paying customers with access to Sotyktu at a cost more than 80% lower than its current list price. This move follows Bristol Myers' recent launch of online sales for its heart drug Eliquis at a reduced price, reflecting a growing trend among pharmaceutical companies to bypass traditional distribution channels.

Manufacturing Expansions and Clinical Trial Updates

Eli Lilly has announced plans for a $6.5 billion manufacturing plant in Houston, Texas, as part of its $50 billion investment in domestic manufacturing. The facility will produce active ingredients for several small molecule drugs, including the experimental obesity medicine orforglipron. Meanwhile, Biogen faced a setback as the FDA rejected a high-dose version of its spinal muscular atrophy drug Spinraza, citing the need for updated technical information related to manufacturing. In other news, Gossamer Bio has secured an option to acquire Respira Therapeutics, a developer of pulmonary arterial hypertension treatments, while Harmony Biosciences reported that its experimental drug for fragile X syndrome failed to meet its primary endpoint in a late-stage trial.