Vanda Pharmaceuticals Inc. will get to take its argument for twice-rejected jet lag disorder drug Hetlioz (tasimelteon) before the U.S. FDA in a formal evidentiary public hearing, a rare move by the agency that the firm claims underscores the “gravity of the legal and scientific issues” it has raised.
A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
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.
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.
The psychedelic and psychedelic-like drug space made another stride as Ataibeckley Inc. disclosed positive top-line data from a double‑blind, placebo‑controlled, first-in-patient phase IIa study testing oral R-MDMA therapy EMP‑01 in adults with social anxiety disorder (SAD).
The effects of aging pose an additional challenge for people with HIV due to the neurological and psychological consequences that persist despite antiretroviral therapy. At the Conference on Retroviruses and Opportunistic Infections (CROI) held Feb. 22-25, 2026, in Denver, the scientific community examined how the virus affects the brain, how the reservoir is established in the CNS, and which genetic, immunological or treatment-related factors influence cognitive health.
Neurosoft Bioelectronics SA has signed an agreement with Science Corp. to gain access to its full stack of clinical-grade neural recording tools as it looks to advance its brain-computer interface (BCI) system.
Vanda Pharmaceuticals Inc. gained U.S. FDA approval for Bysanti (milsaperidone), cleared for use in acute bipolar I disorder and schizophrenia and ensuring continuation of the firm’s atypical antipsychotic franchise, with Fanapt (iloperidone) set to start losing patent protection in 2027. It also offered a rare straightforward regulatory win for Vanda, which has tussled with the FDA in recent years.
Japan is backing conditional approvals of Amchepry (raguneprocel, Sumitomo Pharma Co. Ltd./Racthera Co. Ltd.) and Reheart (Cuorips Inc.), positioning them to become the world’s first induced pluripotent stem cell-derived (iPSC) therapies to receive regulatory clearance.
Med-tech companies with an AI component in their solutions will certainly find investors willing to back them. AI after all, is being used to develop more effective, smarter technologies. However, investors will only deploy capital into innovations that address genuine clinical needs. The aging population is driving interest in devices targeting cardiovascular and musculoskeletal disorders, and other solutions geared toward neurological conditions, women’s health and diagnostics are also attracting investor attention.
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