Earlier this week, the U.S. FDA granted full approval to Moderna Inc.'s COVID-19 vaccine, Spikevax (elasomeran), for adults. Now it's the CDC's turn to consider endorsing the jab. The agency’s Advisory Committee on Immunization Practices (ACIP) heard safety and efficacy data from Moderna at its meeting in Atlanta this morning and will hear an internal analysis of that data later today. In the afternoon, the committee will vote on whether to advise the CDC to formally recommend the vaccine should be given to adults, something that has already been done hundreds of millions of times globally. The Pfizer Inc.-Biontech SE vaccine, Comirnaty (tozinameran), gained full FDA approval on Aug. 23, 2021, for people 16 years of age and older.

Analysis ongoing as top-line data disappoint for Vanda’s gastroparesis drug

Citing potential confounding factors, Vanda Pharmaceuticals Inc. is hoping further analysis might explain the top-line miss for its phase III study testing tradipitant in gastroparesis, hinting that regulatory submissions could still be in the offing. Investors, unsurprisingly, were less optimistic, sending shares of the Washington-based company (NASDAQ:VNDA) down 20% in morning trading. Results showed the phase III study, known as VP-VLY-686-3303, failed to hit its prespecified endpoint, defined as the difference between tradipitant and placebo on the change of the severity of nausea from baseline at week 12. Both arms showed significant improvements from baseline on nausea as well as other symptoms of gastroparesis, a condition involving delayed gastric emptying of solid food despite no obstructions in the stomach.

NICE backs Orchard rare disease drug after further cut to $3.9M list price

In one of its familiar U-turns, the U.K. National Institute for Health and Care Excellence (NICE) has recommended NHS England should fund a rare disease gene therapy from Orchard Therapeutics plc, considered to be the world’s most expensive drug. The list price for Libmeldy (atidarsagene autotemcel) in England and Wales is £2,875,000 (US$3.9 million), making it the most expensive drug that NICE has ever evaluated. The U.K. health technology assessment agency initially rejected funding for the metachromatic leukodystrophy (MLD) medicine in July 2021.

Eisbach Bio surfs synthetic lethal wave

“The premise of our whole company is that we target molecular machines, but we don’t target the engine,” Adrian Schomburg told BioWorld. Instead, “we interfere with the throttle and other highly specific controls of these machines.” “We,” in this case, is Eisbach Bio GmbH, a German startup that is developing anticancer programs aimed at exploiting synthetic lethalities by targeting helicases. Founded in 2019, the company has three programs, a recently announced collaboration with MD Anderson Cancer Center in oncology, and another program in COVID-19.

Social climbers: Bionomics, Vistagen strive for heights in SAD

Bionomics Ltd.’s U.S. debut last December, when the firm raised about $23 million through the sale of American depositary shares, called new attention to the Australian firm and its prospects in social anxiety disorder (SAD) and post-traumatic stress disorder (PTSD). BNC-210, the firm’s lead compound – in phase II trials for SAD as well as PTSD – is a negative allosteric modulator of the alpha 7 nicotinic acetylcholine receptor and bears an up-and-down history.

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AC Immune, Anacardio, Arcellx, Bayer, Biogen, CSL Behring, Cytokinetics, Hemab, Hutchmed, Lactocore, Mainz Biomed, Mallinckrodt, Novavax, Nuvectis, Ocean Biomedical, Pfizer, RDIF, Revolo, Roche Canada, Tevogen, TG, Tiziana, UCB, Valneva, Vanda, Viatris