Becoming the first treatment for rare genetic disorder Prader-Willi syndrome (PWS), breakthrough therapy drug DCCR (diazoxide choline) gained U.S FDA approval as Vykat XR the evening before March 27, its PDUFA date, sending shares of developer Soleno Therapeutics Inc. up 41% in early trading. Soleno’s shares (NASDAQ:SLNO) rose $20.07 to $69.04. In addition to breakthrough therapy, Vykat has orphan drug, fast track and priority review designations in the U.S. The FDA told the company in October 2024 that an advisory committee meeting was not necessary. It is Soleno’s first approved product. Soleno CEO Anish Bhatnagar called it “a significant milestone for the company” and “for the PWS community who have had no options to treat the most disruptive aspect of this disease,” which is hyperphagia, or incessant hunger. Vykat extended-release tablets will be available in the U.S. beginning in April, and a list price of $5.92 per milligram, with the average baseline weight of participants in the C601 study being 61 kilograms, which would cost insurers $466,200 annually.

BIO survey points to ‘damaging impacts’ of Trump tariffs

A survey of members of the Bioindustry Innovation Organization (BIO) has exposed what is at stake for the sector, on both sides of the Atlantic, if proposed tariffs are imposed on pharmaceutical imports to the U.S. "A staggering 94% of biotech firms anticipate surging manufacturing costs if tariffs are placed on imports from the EU,” according to BIO.

HHS to cut staffing levels by 20,000 under efficiency initiative

The U.S. Department of Health and Human Services has announced a plan to reduce staffing by 10,000 in an immediate reduction, which when paired with retirement initiatives will drop staffing by as many as 20,000. HHS Secretary Robert F. Kennedy Jr., said on a LinkedIn video that HHS staffing had increased by 17% during the Biden administration and that HHS has more than 100 communications offices and 40 IT departments, a predicament he said is emblematic of gross inefficiency at HHS.

Equillium plunges on top-line GVHD results with itolizumab

Shares of Equillium Inc. (NASDAQ:EQ) were trading at 51 cents, down 25 cents, or 33%, on top-line data from the phase III Equator study testing itolizumab in first-line treatment of patients with acute graft-vs.-host disease (GVHD). The study results failed to show a meaningful difference in complete response (CR) or overall response rate at day 29 between patients treated with itolizumab and placebo. But statistically significant and clinically meaningful benefit in longer-term outcomes were achieved, including complete response at day 99, duration of CR and failure-free survival. Itolizumab exhibited a favorable safety and tolerability profile and did not increase the risk of clinical sequelae, including infection or sepsis, primary drivers of the high mortality rate associated with acute GVHD. Equillium has been talking with the U.S. FDA and expects feedback during May. If the agency goes along, the company will submit a BLA in the first half of next year.

Bifunctional antibodies gaining ground

Snatching headlines regularly are bispecific antibodies, but the bifunctional approach has gained less press. That could be about to change. Developers aplenty are working with prospects in the category, which involves designing one binding site for an antigen and another site for a non-antibody molecule such as a toxin or drug, taking aim at a single target and bringing a secondary function for added firepower.

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