Uniqure NV is the latest firm to get caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies. The company disclosed in its latest earnings report that U.S. regulators are calling for a sham-controlled study before they will consider approval of gene therapy AMT-130 in Huntington’s disease, a requirement that could set the program back by two to three years and raises potential ethical issues. Read More
The busy Prader-Willi syndrome (PWS) space took another hit as did shares of Aardvark Therapeutics Inc. (NASDAQ:AARD), which closed March 2 at $5.47, down $7.02, or 56%, 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, or intense hunger, in patients with the disease. Read More
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. Read More
Ascendis Pharma A/S’ Yuviwel (navepegritide) gained U.S. FDA accelerated approval Feb. 27 for children 2 and older with the rare genetic bone growth disorder achondroplasia, also known as dwarfism, winning the company a rare pediatric disease priority review voucher Read More
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. Read More
The U.S. CDC’s Advisory Committee on Immunization Practices (ACIP) has two new members, bringing its total membership to 15. As he has done since dismissing the entire ACIP panel last June, Health and Human Services Secretary Robert Kennedy named the new members, Angelina Farella and Sean Downing, barely two weeks before the next ACIP meeting, March 18-19. Read More
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. Read More
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. By phase, the month’s updates consisted of 60 from phase I, 44 from phase II and 40 from phase III. Read More
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. Read More
Biopharma and med-tech companies raising money in public or private financings, including: Boston Scientific, Braincheck, Medx, Penumbra, Zura. Read More
Clinical updates for biopharma and med tech, including data readouts and publications: Adarx, Bayer, Blue Earth, Bluewind, Bonesupport, Curium, Galderma, Immvira, Johnson & Johnson, Organon, OSE, Urogen. Read More
Regulatory snapshots for biopharma and med tech, including global submissions and approvals, and other regulatory decisions and designations: AS Software, Asieris, Boehringer Ingelheim, Deephealth, Eli Lilly, Ipsen, Moderna, Neurogene, Novartis, Optellum, Photocure, Regeneron, Sanofi, Sentynl, Synergy Spine Solutions, X4. Read More
