The U.S. FDA has approved Duvyzat (givinostat), from Italfarmaco SpA, of Milan, Italy, for treating Duchenne muscular dystrophy (DMD). It is the first nonsteroidal drug for treating all of DMD’s genetic variants. The oral treatment is approved for those ages 6 and older. While pricing has not been released by the company, RBC Capital Markets analyst Brian Abrahams wrote that he expects Duvyzat to be priced at a premium akin to currently marketed branded DMD small molecules that range from $100,000 to $150,000 annually. The NDA was filed on the strength of data from the phase III Epidys trial in which those on the drug exhibited a slower decline in their ability to climb four stairs than those on placebo.

Eledon rises on pig-human transplant with tegoprubart

Shares of Eledon Pharmaceuticals inc. (NASDAQ:ELDN) benefited – as did a 62-year-old patient with end-stage renal disease – from the use of tegoprubart, the firm’s investigational anti-CD40L antibody, as part of the immunosuppressive treatment regimen deployed after the first-ever transplant of a kidney from a genetically modified pig to a human. The procedure was done March 16 at Massachusetts General Hospital. The Irvine, Calif.-based firm’s stock was trading at $2.18, up 30 cents, or almost 16%, having risen about 37% during the past five days.

EMA’s CHMP backs new antibiotic, oral PNH drug in March meeting

The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria. If approved, Emblaveo would mark one of the first beta-lactam/beta-lactamase inhibitor combos cleared for use in Europe. The Emblaveo recommendation was one of 12 issued by the CHMP at its March meeting, with the committee also giving a thumbs-up to Novartis AG’s Fabhalta (iptacopan) for use in paroxysmal nocturnal hemoglobinuria (PNH), as well as three biosimilars.

Kazia licenses rare epilepsy drug paxalisib to Korea’s Sovargen

Kazia Therapeutics Ltd. has out-licensed paxalisib as a potential treatment for intractable epilepsy in focal cortical dysplasia type 2 (FCD T2) and tuberous sclerosis complex (TSC) disease in a carve-out deal with Sovargen Co. Ltd. for $20.5 million plus sales royalties. Under terms of the deal, Sovargen of Seoul, South Korea, will develop, manufacture, and commercialize paxalisib as a potential treatment for FCD T2 and TSC disease. In exchange, Sydney-based Kazia will receive an up-front payment of $1.5 million, and $19 million in development and regulatory milestones, plus sublicensing revenues and royalties on net sales for products incorporating paxalisib. The licensing agreement includes all countries worldwide, excluding mainland China, Hong Kong, Macao and Taiwan, where Kazia retains rights.

US CBO: Numbers don’t add up yet for Part D obesity drug coverage

When it comes to whether Medicare Part D should cover the new anti-obesity drugs, the U.S. Centers for Medicare & Medicaid Services and lawmakers may be caught between the math and public pressure. With their current monthly prices ranging from $1,100 to $1,300, weight-loss drugs like Novo Nordisk A/S’ Wegovy (semaglutide) and Eli Lilly and Co.’s Zepbound (tirzepatide) “would cost the federal government more than it would save from reducing other health care spending — which would lead to an overall increase in the deficit over the next 10 years,” the Congressional Budget Office reported this week. Meanwhile, lawmakers, doctors and other stakeholders are pushing to do away with the Part D ban that prohibits Medicare coverage of drugs to treat obesity.

CMS: Coverage of obesity drug Wegovy for cardiovascular events OK

Following the U.S. FDA’s March 11 approval of Novo Nordisk A/S’ glucagon-like peptide 1 receptor agonist Wegovy (semaglutide) to reduce the risk of major adverse cardiovascular events such as heart attack and stroke, the Centers for Medicare & Medicaid Services (CMS) has issued guidance ensuring coverage. The guidance stated that anti-obesity medications that are approved “for an additional medically accepted indication can be considered a part D drug for that specific use,” a CMS spokesperson told BioWorld. That means Wegovy will now be covered, but only for obese and overweight individuals who also have “established cardiovascular disease.”

11 US approvals in February include biosimilar, treatments for frostbite, HIV

In February, the U.S. FDA greenlit 11 new drugs, a slight decrease from the 14 approvals seen in the same month the previous year but up from eight FDA approvals in January. Throughout 2023, the FDA maintained a robust pace, granting an average of nearly 16 approvals per month, an uptick from 2022’s monthly average of 12.5 approvals.

What’s going on beyond amyloid-β in Alzheimer’s disease management

After many years of testing different monoclonal antibodies against amyloid-β protein, the results obtained are far from being outstanding, and the control of the progression and symptoms of Alzheimer’s disease (AD) remains elusive. At the recent AD/PD 2024 conference held in Lisbon, new non-anti-amyloidogenic strategies in the starting line against AD were discussed. Professor Einar Sigurdsson from New York University gave a presentation entitled, “Single domain antibodies for therapy and diagnosis of synucleinopathies and tauopathies.” The key advantage over whole antibodies is that single-domain antibodies have a better blood-brain-barrier permeability because of their small size, around 13 kDa.

Also in the news

Abbvie, Adocia, Bergenbio, Bristol Myers Squibb, Camp4, Eisbach, Graphite, Hi-Bio, IGC, Inhalon, Johnson & Johnson, Kazia, Lenz, Lisata, Maat, Merck, Mission, Morphosys, Nika, Nkarta, Novartis Nykode, Orion, Oscotec, Otsuka, Parvus, Phanes, Public Health Vaccines, Sangamo, Scilex, Sosei Heptares, Sovargen, Theratechnologies, Vertex