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

In a week marked by significant developments across the pharmaceutical landscape, regulators have greenlit new treatments, government agencies have launched initiatives to accelerate gene therapy development, and major players have announced strategic moves in manufacturing and drug pricing.

Crinetics Challenges Industry Giants with Acromegaly Drug Approval

Crinetics Pharmaceuticals has secured FDA approval for Palsonify (paltusotine), the first once-daily oral therapy for acromegaly, a rare hormonal disorder. The drug is approved for adults 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.

Federal Initiatives Aim to Accelerate Gene Therapy Development

The U.S. government has unveiled multiple initiatives to foster gene therapy research and development. The Food and Drug Administration (FDA) has published draft guidance outlining accelerated approval pathways, post-approval requirements, and new clinical trial designs for gene therapies. Simultaneously, the Advanced Research Projects Agency (ARPA) has launched two programs aimed at speeding development and improving manufacturing processes to reduce production costs for these complex medicines.

Bristol Myers Squibb and Eli Lilly Make Strategic Moves

Bristol Myers Squibb (BMS) is set to begin selling its psoriasis drug Sotyktu online at a discounted price starting January 2026. The initiative will offer the medication at more than 80% below its current list price for uninsured, underinsured, or out-of-pocket paying customers. This move follows BMS's recent offering of its heart drug Eliquis online at a reduced price, signaling a growing trend among pharmaceutical companies to provide products directly to consumers while bypassing traditional distribution channels.

In manufacturing news, Eli Lilly has announced plans for a $6.5 billion factory in Houston, Texas, which will produce active ingredients for several small molecule drugs, including its experimental obesity medicine orforglipron. The facility is expected to employ 4,000 people during construction and 615 once operational in five years. This announcement is part of Lilly's broader $50 billion commitment to domestic manufacturing, which includes another recently announced plant near Richmond, Virginia.

Setbacks and Acquisitions in Drug Development

Biogen faced a setback as the FDA rejected a high-dose version of its spinal muscular atrophy drug Spinraza. The agency did not cite deficiencies with clinical data but requested an update to certain technical information related to manufacturing. Biogen plans to respond promptly, though the timeline for potential approval remains unclear.

Meanwhile, Gossamer Bio has secured an option to acquire Respira Therapeutics, a privately held company developing treatments for pulmonary arterial hypertension. The deal provides Respira with funding to advance its lead candidate, RT234, an inhaled drug that recently completed a Phase 2b study.

In another development, Harmony Biosciences reported that its experimental drug ZYN002, a topical synthetic cannabidiol for fragile X syndrome, failed to meet its primary endpoint in a late-stage trial due to a higher than expected placebo response rate.

As the pharmaceutical industry continues to evolve, these developments highlight the ongoing challenges and opportunities in drug development, regulatory approval, and market strategies across various therapeutic areas.