→ Ul­tragenyx’s Crysvi­ta has scored its sec­ond FDA ap­proval, earn­ing the nod to treat bone soft­en­ing in pa­tients with the ul­tra-rare dis­ease known as tu­mor-in­duced os­teo­ma­la­cia. First ap­proved in 2018 for a dif­fer­ent ul­tra-rare in­di­ca­tion, x-linked hy­pophos­phatemia, Crysvi­ta quick­ly earned watch­dog scorn for its $200,000 per year price tag. De­spite hopes, though, that the price would bring block­buster sales, it earned on­ly $87.3 mil­lion last year and $28.8 mil­lion in Q1 2020. The drug is al­so mar­ket­ed by Japan­ese biotech Ky­owa Kirin.

→ Sanofi and Re­gen­eron’s IL-3/IL-14 block­buster Dupix­ent has been ap­proved in Chi­na for atopic der­mati­tis. CEO Paul Hud­son said that as part of the gov­ern­ment’s Healthy Chi­na 2030 ini­tia­tive, the French phar­ma will seek 25 new ap­provals by 2025.

→ Sagimet Bio­sciences re­leased pos­i­tive Phase II da­ta for its NASH drug TVB-2640. Al­though the field has strug­gled de­spite great in­vestor and in­dus­try promise, most re­cent­ly with the fail­ure of Gen­Fit’s close­ly-watched Phase III tri­al, a steady pipeline of ear­li­er stage drugs con­tin­ue to march along. Sagimet, known as 3-V Bio­sciences un­til last Au­gust, said the drug’s high dose had a 61% re­sponse rate across 30 pa­tients and led to a mean liv­er fat re­duc­tion of 28.2%.

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).