Barely a year after the U.S. FDA shackled Abeona Therapeutics Inc.’s cell-based gene therapy with a complete response letter, the agency has approved it for treating a rare and genetic skin disease. Zevaskyn (prademagene zamikeracel), for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), will be priced in the U.S. at $3.1 million. The company told BioWorld the price is “directly tied to recognizing the value for a ground-breaking gene therapy designed to deliver long-lasting wound healing and pain reduction after a single treatment application, even in the most troublesome, tough large, chronic RDEB wounds.” The therapy is expected to be available sometime in the third quarter of 2025.

Stealth PDUFA date delivers another disappointing delay

Stealth Biotherapeutics Inc., and, more importantly, patients with Barth syndrome, faced another disappointing delay today when the U.S. FDA kicked its approval decision down the road for Stealth’s elamipretide. April 29 was the second PDUFA date the FDA had set for the drug, which has been in development for more than a decade to become the first approved treatment for the ultra-rare mitochondrial disease that currently affects fewer than 150 males in the U.S. and fewer than 300 worldwide. In announcing the latest delay, Stealth CEO Reenie McCarthy noted that the FDA has confirmed postmarketing requirements for the drug and recently initiated labeling discussions.

Regeneron earnings report includes Eylea slip, CRL

Wall Street clipped Regeneron Pharmaceuticals Inc. shares (NASDAQ:REGN) by $45.71 to $565.14 after the firm disclosed financial results for the first quarter of 2025 and provided a business update. Sales of age-related macular degeneration treatment Eylea (aflibercept) fell short of expectations, and the U.S. FDA issued a complete response letter regarding the prefilled syringe for Eylea HD. Biosimilar competition has become a problem for the drug, too.

Orum halts US study of DAC cancer asset after patient death

Orum Therapeutics Inc. on April 26 pulled the plug on a U.S.-based phase I study of ORM-5029, its lead oncology degrader antibody conjugate (DAC) asset, a decision that came months after the company first reported a patient death in November 2024. Orum initially gained U.S. FDA clearance in 2022 for the first-in-human phase I study testing ORM-5029 as a treatment for HER2-positive metastatic breast cancer and other HER2-expressing malignancies or solid tumors.

Ono terminates development of Chordia’s cancer drug candidate

Ono Pharmaceutical Co. Ltd. terminated development of CTX-177 (ONO-7018), its ex-oncology candidate in-licensed from Chordia Therapeutics Inc. in December 2020 for up to ¥52.9 billion (US$370.37 million). CTX-177 is a mucosa-associated lymphoid tissue lymphoid tissue lymphoma translocation protein 1 (MALT1) inhibitor in a phase I study as a potential therapy for advanced non-Hodgkin's lymphoma or chronic lymphocytic leukemia.

AACR 2025: Extra(chromosomal) DNA gives boost, but also vulnerability, to tumors

“I think we’ve come a long way in understanding the importance of this biology. We know it affects men and women, children and adults,” Paul Mischel told the audience during his plenary talk at the 2025 Annual Meeting of the American Association for Cancer Research. The biology Mischel was referring to is that of extrachromosomal DNA or ecDNA – circular DNA that can be present in cancer cells and has strong effects on tumor biology when it is.

100 days of uncertainty

Massive terminations. Axed programs. Canceled grants and contracts. Communication freezes. Threats and more threats of tariffs. Lawsuit upon lawsuit. Policy whiplash. The first 100 days of the Trump administration have been nothing short of chaotic, both in the U.S. and throughout the world. Shining a light through the uncertainty, BioWorld continues to cover the administration’s latest policy decisions and actions affecting the life sciences sector, as well as their impacts across the globe.

Also in the news

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