Syphilis drug shortage turns preventable diagnoses real

A pregnant woman in rural Arizona with syphillis could have been treated fairly easily with an injection of penicillin. But the drug, Bicillin L-A, is manufactured only by Pfizer, and has been in short supply for a year now — which meant the drug arrived too late to prevent probable congenital syphilis in her newborn.

The syphilis epidemic is worsening, congenital syphilis cases are up roughly 800% since 2012 — and this Bicillin shortage is expected to continue through 2027. Public health officials say Pfizer’s emergency allocation system is confusing and may disadvantage health departments serving low-income patients.

A box of 10 syringes can cost around $8,000, and the drug is manufactured at only one Pfizer plant in Michigan to supply the whole country.

“Most shortages are of not-very-profitable drugs,” said Erin Fox, associate chief pharmacy officer at University of Utah Health.

diabetes

FDA approves Sanofi drug for stage 3 type 1 diabetes

The FDA has approved Sanofi’s Tzield for children 8 and older with stage 3 type 1 diabetes, expanding the drug’s use. The application was reviewed under former Commissioner Marty Makary’s expedited National Priority Voucher program. But it missed its target decision date after then-top drug regulator Tracy Beth Høeg reportedly challenged staff recommendations to approve the therapy. She was apparently concerned about risks including Epstein-Barr virus reactivation and potential cancer links.

Tzield, or teplizumab, is a monoclonal antibody that aims to delay the progression of type 1 diabetes. The FDA first approved the drug to delay the onset of stage 3 diabetes in adults, and in children ages 8 and older with stage 2 disease. In April, the FDA expanded that approval to allow children as young as 1 year old to take the medication.

drug pricing

Trump targets Medicare negotiation loophole

The Trump administration wants to change Medicare’s drug price negotiation rules: It doesn’t want drugmakers to extend being protected from the negotiation process by adding new ingredients to existing medicines. The proposal would specifically target biologics reformulated with hyaluronidase, an enzyme that allows drugs traditionally delivered through lengthy infusions to be administered as quick subcutaneous injections.

Under the plan, certain reformulated products would still be treated as the same underlying drug for Medicare negotiation purposes rather than receiving a fresh exemption period. CMS argues the change is necessary to preserve the intent of the Inflation Reduction Act and prevent companies from sidestepping negotiation timelines. But drugmakers say that these combination products represent meaningful innovation and should qualify as new medicines.

cancer

Lilly's Ajax acquisition may have been worth it

Eli Lilly unveiled the first clinical data behind its up-to-$2.3 billion acquisition of Ajax Therapeutics, FierceBiotech writes. The results suggest the company may now have in its arsenal a serious challenger to Incyte’s dominance in myelofibrosis, a rare blood cancer. In an early-stage trial, Ajax’s type II JAK2 inhibitor, AJ1-11095, reduced spleen volume in 70% of heavily pretreated patients. It improved symptoms dramatically, and seemed to reduce the underlying disease-driving mutations across multiple genetic subtypes — hinting it might offer disease-modifying effects.

“This medicine started working right out of the gate, with a good safety profile,” oncology and business development head Jake Van Naarden told Fierce.

Though the study remains small and early, the results perhaps explain why Lilly was willing to spend billions on Ajax.

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