The latest firm caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies, Uniqure NV saw its shares plummet after disclosing that U.S. regulators are requiring a sham-controlled study before considering approval of gene therapy AMT-130 in Huntington’s disease, a move that could set the program back by two to three years. Uniqure said the FDA noted data from the firm’s phase I/II studies, which compared AMT-130 to an external control, were not sufficient for supporting a marketing application. The design for a randomized, double-blind, sham surgery-controlled study will still need to be determined, including safety and ethical implications, and company executives said they plan to request a type B meeting with the FDA in the second quarter.

Aardvark stock plunges on phase III pause in Prader-Willi syndrome

Shares of Aardvark Therapeutics Inc. (NASDAQ:AARD) were trading at $5.52, down $6.97, or 55%, after the company disclosed a voluntary pause of the phase III Hunger Elimination or Reduction Objective (HERO) trial testing ARD-101 as a treatment for hyperphagia in patients with Prader-Willi syndrome (PWS). The HERO study, as well as the open-label extension effort, are held up due to reversible cardiac observations at above-target therapeutic doses found during routine safety monitoring in a healthy volunteer study. San Diego-based Aardvark has nixed the planned third-quarter report of top-line HERO data in PWS, saying further guidance should be available in the second quarter. ARD-101 is a gut-restricted small molecule agonist of select taste receptors expressed on the luminal side of the intestine.

NICE recommends leadless pacemakers to treat slow heart rhythms

The U.K.’s National Institute for Health and Care Excellence (NICE) recommendation that leadless cardiac pacemakers be used as the standard of care for people with slow heart rhythms (bradyarrhythmias) is a boon for more than 2 million individuals living with the condition. The use of the technology will transform patients’ quality of life, reduce rates of infections and lower costs for the health care system.

Dwarfism drug gets FDA accelerated approval for Ascendis

Ascendis Pharma A/S’ Yuviwel (navepegritide) gained U.S. FDA accelerated approval Feb. 27 for children 2 and older with the genetic bone growth disorder achondroplasia, also known as dwarfism, winning the company a rare pediatric disease priority review voucher. Yuviwel, a prodrug of C-type natriuretic peptide (CNP) and developed as Transcon CNP, is the first and only once-weekly treatment aimed at increasing linear growth in those with open epiphyses. It is also the only treatment that provides continuous systemic exposure of active CNP to tissue receptors throughout the body, counteracting the overactive FGFR3 signaling in achondroplasia.

HITting solid tumors with a closer look and a stronger CAR T cell

CAR T cells have been groundbreaking for the treatment of B-cell cancers. But 8 years after Kymriah (tisagenlecleucel, Novartis AG) became the first CAR T-cell therapy to be approved, there are no CAR Ts approved for solid tumors. There are several reasons that solid tumors are harder for CAR T cells to get a handle on. One of the big obstacles is “finding that appropriate antigen … that sweet spot where you have expression on all your tumor cells, but not also on healthy tissues,” Sophie Hanina told BioWorld. Hanina is a postdoctoral fellow at Columbia University who authored work published in Science on Feb. 26, 2026, indicating that CD70, which is expressed on more than 20 types of solid tumors, may be such an antigen.

ACIP grows by two ahead of next meeting

The U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) has two new members, bringing its total membership to 15. As he has done from the beginning, Health and Human Services Secretary Robert Kennedy named the new members – pediatrician Angelina Farella and Sean Downing, a doctor specializing in internal medicine and pediatrics – just two weeks before ACIP’s next meeting, March 18-19. And as in the past, at least one of the appointments is raising more than eyebrows.

Adquey finds hatchway, still more in PDE4

The U.S. FDA clearance Feb. 20 of East Windsor, N.J.-based Acrotech Biopharma Inc.’s PDE4 inhibitor Adquey (difamilast ointment 1%) for mild to moderate atopic dermatitis brought renewed focus on the mechanism, where other drugs are already approved and more are stocking developers’ pipelines across indications. PDE4 activity centers on increasing intracellular cyclic adenosine monophosphate, a key second messenger that regulates inflammatory responses. Top names in the space include Amgen Inc., Pfizer Inc., Boehringer Ingelheim GmbH and Arcutis Biotherapeutics Inc.

Phase III wins lift Alumis, Corcept shares in January

BioWorld tracked 144 clinical trial readouts across phases I through III in January 2026, down from 215 in December. January included 15 phase III trials reporting positive results and three that failed to meet key endpoints. With six months remaining until the July 11, 2026, PDUFA date for relacorilant in platinum-resistant ovarian cancer, Corcept Therapeutics Inc. reported a 35% reduction in mortality risk, meeting the overall survival endpoint after achieving statistical significance in progression-free survival last March. And a potential psoriasis contender is emerging in the competitive oral tyrosine kinase 2 inhibitor landscape, as shares of Alumis Inc. (NASDAQ:ALMS) surged 95% to close at $16.23 on Jan. 6, after reaching an intraday high of $22.30, following positive phase III Onward1 and Onward2 results for envudeucitinib.

Med-tech outlook 2026: exits, financings, headwinds

What does med tech in 2026 look like? Uncertainty remains. Global investors are confident about exit opportunities in the year ahead. But financing will be targeted. For the med-tech companies, geopolitical tensions continue to create headwinds, particularly for the European firms navigating an uncertain macroenvironment created by the reciprocal tariffs on goods entering the U.S., their primary market. By contrast, U.S. companies are bullish and believe they are well-positioned to navigate macroeconomic pressures. Still, across both regions, there is confidence in the sector and companies remain focused on advancing their technologies to address clinical needs – innovating, conducting trials, presenting data, raising funds, and delivering products that improve patients’ lives.

Also in the news

Adarx, AS Software, Asieris, Bayer, Blue Earth, Bluewind, Boehringer Ingelheim, Bonesupport, Boston Scientific, Braincheck, Curium, Deephealth, Dexcom, Eli Lilly, Galderma, Immvira, Ipsen, Johnson & Johnson, Lohmann, Matricelf, Medx, Moderna, Monod, Neurogene, Novartis, Optellum, Organon, OSE, Penumbra, Photocure, Regeneron, Sanofi, Sentynl, Synergy Spine Solutions, Tidewave, Urogen, X4, Zura