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• The FDA approved Cablivi (caplacizumab-yhdp) for injection for treatment of pediatric patients aged 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP), to be used in combination with plasma exchange and immunosuppressive therapy.

• The approval expands the indication for Cablivi, which was initially approved in 2019 for the treatment of adults with aTTP.

• aTTP is a rare, life-threatening thrombotic microangiopathy characterized by widespread platelet-rich microthrombi in small blood vessels.

• Although the condition is most often diagnosed in adults, pediatric cases do occur and are associated with substantial morbidity. The estimated annual incidence of aTTP in children is approximately 1 per 10 million. (Contemporary Pediatrics)

• The FDA approved Aqvesme (mitapivat) to treat thalassemia, following a three-month delay and a missed deadline.

• The approval marks a key step forward for Agios as it looks to expand the drug’s use in blood disorders. Mitapivat was first approved as Pyrukynd in 2022 for adults with PK deficiency, and Agios plans on filing an FDA submission next year in sickle cell disease despite mixed phase 3 results.

Together with thalassemia, Agios has said the three indications have “multibillion-dollar potential.” (Endpoints)

• The FDA approved Yartemlea (narsoplimab-wuug) for transplant-associated thrombotic microangiopathy (TA-TMA), making it the first therapy specifically cleared for the life-threatening complication of haematopoietic stem-cell transplantation.

• Driven by activation of the complement system’s lectin pathway, the condition can develop after any stem-cell transplant, but it is more common following allogeneic procedures than autologous ones.

• Recent studies suggest TA-TMA strikes up to 56% of allogeneic transplant recipients; roughly 30,000 allogeneic procedures occur annually across the US and Europe. (FirstWord Pharma)

• The FDA approved Nereus (tradipitant), an oral neurokinin-1 (NK-1) receptor antagonist, to prevent vomiting induced by motion in adults.

• The approval marks the first new drug treatment for motion sickness in over four decades and signifies an advancement in understanding motion sickness mechanisms, Vanda said.

• Motion sickness is thought to be caused by sensory conflict when the brain receives different signals about motion from visual, vestibular, and proprioceptive inputs. This conflict triggers the release of substance P, a key neurotransmitter, and activates NK-1 receptors in the central nervous system (CNS), leading to nausea and vomiting. Tradipitant blocks this pathway by selectively targeting NK-1 receptors.(MedPage Today)

• Health officials’ decision to downgrade a half dozen vaccines on the federally recommended childhood schedule has drawn condemnation across the pharmaceutical sector, with some of the largest drugmakers in the world arguing the move has little to no rationale.

“This shift away from a safe, effective and proven childhood schedule puts our entire pediatric population at significant risk,” a Sanofi spokesperson said.

Merck, which had more than one product impacted, called the decision “a concerning departure from the high-standard science that has guided health authorities and positioned the US as a leader in public health.”

• The move also sets up a fresh debate over Merck’s HPV vaccine Gardasil, which has been a significant moneymaker for the company and is one of the most effective vaccines available against the disease. (Endpoints)

• GSK and Ionis Pharmaceuticals’ antisense oligonucleotide bepirovirsen achieved a functional cure in a pair of late-stage chronic hepatitis B studies, clearing the way for a regulatory submission this quarter.

• Without revealing specific data, GSK said that bepirovirsen met its primary efficacy endpoint in the phase 3 B-Well 1 and B-Well 2 trials, eliciting a “statistically significant and clinically meaningful functional cure rate” in treated patients.

  • The studies defined “functional cure” as having no detectable levels of virus in the blood, as measured by the hepatitis B surface antigen and viral DNA for 24 weeks after a “finite course of treatment.” (BioSpace)

• Basilea Pharmaceutica is continuing its quest to develop much-needed new antifungal drugs by penning a deal with Prokaryotics.

• The collaboration will see Switzerland-based Basilea and US-based Prokaryotics work together to develop a first-in-class broad-spectrum antifungal to treat invasive infections caused by strains of Candida, Aspergillus and rare molds.

• Once a suitable candidate reaches the clinic, Basilea will take charge of further development, according to the Jan. 7 release.

Basilea Chief Scientific Officer Laurenz Kellenberger, Ph.D., said there “remains a significant unmet medical need for safe, effective, and easy to administer antifungals, with activity against priority pathogens.” (Fierce Biotech)

• Overall, the leaders espoused optimism around the thawing of investment dollars flowing to biotechs, as well as the strength of the fundamental principles undergirding the industry, despite whatever curveballs the current administration or its new-look FDA might throw.

• The leaders also rallied around the importance of companies honing their manufacturing chops, regardless of the tariff environment, and touched on the continued impact of direct-to-patient drug sales channels.

• The FDA approved the pill for obesity on Dec. 22.

• It’s also approved to reduce the risk of heart attack and stroke in patients who are obese or overweight.

• The pill follows the blockbuster success of Novo Nordisk’s Wegovy injection, which has been on the market since 2021 and became so popular that it was in short supply until February 2025.

• In a study published in the New England Journal of Medicine, a 25 milligram Wegovy pill led to a 13.6% reduction in weight on average over 64 weeks.

• Patients taking a placebo in the study lost 2.2% of their weight.

• For patients who stayed on the treatment, reduced their calorie intake and exercised, Novo Nordisk estimates they would have a 16.6% reduction in their weight.

• The most common side effects with the Wegovy pill are similar to the injection and include nausea, diarrhea, and vomiting. (NPR)