The U.S. FDA has approved Zycubo (copper histidinate, CUTX-101) as the first treatment for Menkes disease, a rare, genetic disease affecting children who cannot absorb copper through their intestines, leading to seizures, weak muscles, a failure to thrive and, ultimately, if left untreated, an early death by age 3. The approval brought a rare pediatric disease priority review voucher (PRV) to the NDA sponsor, Sentynl Therapeutics Inc., which took over development and commercialization duties in 2023 from Cyprium Therapeutics Inc., a subsidiary of Fortress Biotech Inc. Under that agreement, the PRV will be transferred to Cyprium, which is also eligible for tiered royalties and up to $129 million in milestone payments. The approval arrived two days before the Jan. 14 PDUFA date and followed a complete response letter related to manufacturing issues last fall.

JPM week: Panelists offer dealmaking forecast for 2026

At the Biotech Showcase 2026 – part of the broader hoopla in San Francisco surrounding the J.P. Morgan 2026 Healthcare Conference – a panel of investors and another of pharma dealmakers discussed what to expect from drug company deals in the year ahead. The experts shared everything from whether it’s a buyers’ or sellers’ market to whether the uncertainty is calming down.

Cell/gene therapy sector now sustainable; China competition mounting

There was an upbeat message for cell and gene therapy companies in the 2026 industry update presented as the J.P. Morgan Healthcare conference opened on Monday, with Tim Hunt, CEO of the Alliance for Regenerative Medicine, telling delegates that after lean years of learning, adapting and setbacks, the sector is now self-sustaining. That is quite a change from a year ago, when Hunt painted a glass half full picture.

Pace of EOs slows, but still wields big impact

Perhaps the biggest indicator of U.S. President Donald Trump’s activism in his second term is the 225 executive orders (EOs) he issued in 2025. The pace of those orders seems to have slowed, with “only” 16 released in the last quarter of the year. Four of the recent EOs could impact drug and device companies in a myriad of ways, as they could affect hiring at the FDA, elevate the development and use of artificial intelligence in health care, crack down on foreign-owned and activist proxy advisers that use the shareholder voting process to shape the policies and priorities of America's public companies, and finally end the federal stonewalling that’s prevented a lot of research into the potential medical uses of marijuana.

Oricell raises $70M series C to advance China-developed CAR Ts

Oricell Therapeutics Holdings Ltd. announced a $70 million series C1 round to expand its global footprint and speed clinical development of its CAR T therapies. The round was co-led by Beijing Medical and Health Care Industry Investment Fund, Qiming Venture Partnersa and a leading global health care fund. “As an early investor in Oricell, we've witnessed the company's evolution from a promising startup to a technology powerhouse, from advancing autologous CAR Ts to secreting CAR Ts, to pioneering a three-day rapid manufacturing process, and now to exploring in vivo CAR T in the clinic,” commented Qiming Ventures Managing Partner Xubo Hu.

ACIP expands again, but no new infectious disease specialists

U.S. Health and Human Services Secretary Robert Kennedy has once again expanded the CDC’s Advisory Committee on Immunization Practices (ACIP), this time adding two more OB-GYNs to the membership list. As a result, the ACIP, which can have up to 19 members, now numbers 13, three of which are OB-GYNs. In comparison, only two members are recognized experts in infectious diseases, whereas at least seven of the 17-member panel Kennedy terminated in June had specialties in infectious diseases and epidemiology.

Also in the news

Actuate, Alloy, Alumis, Amplia, Aptevo, Arcadia, Atara, BCM, Benitec, Bioage, Bioxcel, Bit.bio, BMS, Bright Minds, Camurus, Cyotherx, Dogwood, Domain, Eikon, Eisai, Encoded, Erasca, Eureka, Fabentech, Foghorn, Formosa, Gradalis, Helsinn, Idorsia, In8bio, Johnson & Johnson, Kinaset, Lakewood-Amedex, Lexeo, Link Cell, Lipocine, Lynk, Lyra, Medicus, Metagenomi, Mirador, Novita, Nuvation, Obsidian, Ocugen, Oncolytics, Oruka, OS, Pfizer, Phanes, Pharmaessentia, Radius Health, Recludix, Regenxbio, Royalty, Samil, Sanofi, Sebela, Summit, Swiss Rockets, Teva, Vaximm, Veradermics, Vibrant, Viking, Xoma, Zymeworks

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