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• The FDA has given the go-ahead to Ascendis Pharma’s Yuviwel (navepegritide) to boost growth in kids 2 years and older with achondroplasia.

• Yuviwel is the first drug on the market to use weekly dosing to provide sustained systemic exposure to C-type natriuretic peptide (CNP), a key signaling molecule affected in achondroplasia, according to Ascendis’ news release.

• Achondroplasia is a genetic disease characterized by faulty bone development in children. Though classified as a rare disease, achondroplasia is the most common cause of dwarfism. Navepegritide, Yuviwel’s active ingredient, addresses a key disease pathway by providing continuous exposure to CNP, thus promoting bone growth. (BioSpace)

• The FDA approved Palynziq (pegvaliase-pqpz) for pediatric patients aged 12 years and older, providing a new therapeutic option for adolescents struggling with PKU management.

• Results from the phase 3 PEGASUS trial demonstrated significant reductions in blood phenylalanine (Phe) levels compared to diet alone, with many patients achieving levels below guideline recommendations.

• Unlike traditional management that requires an ultra-restrictive diet, this treatment may allow patients to increase natural protein intake while maintaining safe Phe levels. (Contemporary Pediatrics)

• Novo Nordisk received approval from the FDA for three new indications for once‑weekly growth hormone Sogroya, expanding its use to children aged 2.5 years and older with idiopathic short stature, those born small for gestational age without catch‑up growth by age two, and those with growth failure linked to Noonan syndrome.

• The company said the decision broadens treatment options for families and clinicians while offering an alternative to daily injections, which can be difficult to maintain. (PharmaTimes)

• The FDA approved Juxtapid (lomitapide) capsules for children as young as 2 years of age with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that causes extremely high levels of low-density lipoprotein cholesterol (LDL-C).

• Without early intervention, those living with this condition face an increased risk of severe atherosclerosis.

• Juxtapid is only available through a risk evaluation and mitigation strategy (REMS) program, and it is delivered by specialty pharmacy partner Accredo.

• Juxtapid has a black box warning because of a risk related to liver injury; it can cause increased liver enzymes or increased fat in the liver.

• Taking Juxtapid requires that patients maintain a low-fat diet. The company offers a program called Chiesi Total Care that is staffed by dietitians to provide support to patients in this area and to help navigate insurance. (Managed Healthcare Executive)

• A Boehringer Ingelheim drug now has FDA approval as an earlier treatment for lung cancers expressing a certain genetic signature, a regulatory decision handed out under the agency’s new Commissioner’s National Priority Review Voucher (CNPV) pilot program to accelerate the review of medicines deemed important to public health or national security.

• The drug, Hernexeos (zongertinib) is an oral small molecule inhibitor of a protein called HER2.

• Last August, the FDA granted accelerated approval for the once-daily tablet as a second-line treatment for advanced cases of non-small cell lung cancer (NSCLC) with HER2 mutations.

• The regulatory decision permits use of the drug as a first-line treatment. (MedCity News)

• Moderna has agreed to pay up to $2.25 billion to Arbutus Biopharma and Roivant subsidiary Genevant Sciences in a settlement that ends a longstanding legal dispute over technology used in its mRNA vaccines.

• In a statement, Moderna said it will make an upfront payment of $950 million in the third quarter of this year while appealing another $1.3 billion in possible payouts to a federal circuit court. Moderna won’t have to pay future royalties on sales of its mRNA vaccines following the initial lump sum payment.

• The settlement comes just days before the scheduled start of a jury trial in Delaware and leaves Moderna liable for far less than the roughly $5 billion Arbutus and Genevant had been seeking. (BioPharma Dive)

• Shares in uniQure were down about 35% on March 2 after the company revealed that the FDA hasn’t budged on its stance that phase I/II data of its Huntington’s disease gene therapy AMT-130, compared against an external control, aren’t sufficient for approval.

• The feedback, outlined in minutes from a Type A meeting in January, reiterates the FDA’s recommendation that the company run a new trial. (FirstWord Pharma)

• Generate:Biomedicines, a leader of the next wave of AI-driven biotechs, completed one of the industry’s largest IPOs in years, although the milestone was tarnished by a rough first few hours of trading for the new stock.

• Earlier February 27—minutes after ringing Nasdaq’s opening bell and surrounded by several dozen co-workers—Generate:Biomedicines CEO Mike Nally spoke about the Somerville, MA-based biotech’s plans for using its raise of about $400 million.

• Much of it will pay the bills for typical drug development, bankrolling phase 3 studies for its lead program, a TSLP-targeting antibody that is being studied in asthma. Nally credited that program with helping Generate through a speedy IPO process. (Endpoints)

• On February 25, Lilly leaders, including Diogo Rau, chief information and digital officer, and Thomas Fuchs, PhD, Chief AI Officer, cut the ribbon on a new Nvidia supercomputer, named LillyPod, that the Big Pharma believes to be the most powerful in the industry.

• After first announcing the supercomputer in October, Lilly and Nvidia followed up with a $1 billion commitment for a new Bay Area AI co-innovation lab, announced at the January J.P. Morgan Healthcare Conference in San Francisco.

• LillyPod will help the Indianapolis drugmaker speed up development timelines, Rau said, but in piecemeal ways. By automating clinical trial tasks like patient enrollment and optimizing manufacturing processes, he hopes Lilly can cut the typical 10-year timeline for a new drug down to five years.

• In terms of manufacturing, Lilly uses AI “all over the place” in its production process. For example, an auto-injector—like those used for weight loss blockbuster Zepbound—can be photographed 70 or 80 times in a “split second” and analyzed for defects by AI.

• “We’ve also used AI extensively in forecasting,” Rau added, which has greatly improved the drugmaker’s supply and demand balance.

• But there is one area where humans will always win out over AI, Fuchs chimed in: curiosity. (Fierce Biotech)

• Plotkin, now 93, has seen deadly childhood disease after deadly childhood disease quelled by vaccines over his lifetime. It just so happens that he had a hand in developing a number of them, including the vaccines that protect against rubella and rotavirus.

“All I can say is that I’m beginning to regret having lived so long—because we’re going downhill,” Plotkin said.

“And I think that’s one reason why Stanley is so discouraged right now. Because he’s seeing his life’s work dismantled, in some cases, repudiated, on specious grounds,” Straus said in an interview.

“He is one of a handful of people who were very heavily involved, at the individual scientific level, in developing vaccines. I mean, if you look at the cohort back then, there’s Stanley Plotkin, there’s Maurice Hilleman, there’s Hilary Koprowski,” Fauci said. (Hilleman was involved in the development of multiple vaccines and died in 2005; Koprowski developed a rabies vaccine, among others, and died in 2013.)

“He was there when it was bad and he helped to make it good,” he said. (STAT)