BioSpace News Archive

The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.

Biogen is growing as planned, with marketed products generating more revenue and multiple late-stage readouts expected over the next year. Building out an early-stage pipeline would be a bonus, CEO Chris Viehbacher said on a Q3 earnings call Wednesday.

Three-country commercial framework value of approximately US$550,000 over its initial three-year term based on VIVI Cap pricing at the scheduled minimum unit volumes Highlights Definitive exclusive distribution agreement with SPORTORO d.o.o. for Croatia, Serbia and Bosnia and Herzegovina. Commercial scope covers VIVI Cap™, VIVI Epi™ and VIVI Med™, extending access across insulin pens, epinephrine auto-injectors, vials and other compatible temperature-sensitive medication formats. The agreement contemplates the initial order is to be placed and fully paid in accordance with the agreement, with the second targeted within six months. At exact minimum unit targets and current all-VIVI Cap pricing tiers, indicative three-year product purchases would be a minimum of approximately US$550,000; realized value depends on product mix, volumes, approvals and performance with future upside potential. SPORTORO brings Croatian consumer-products and e-commerce experience, providing an entry point for developing medication-protection sales channels across three Southeast European markets. TempraMed now has distribution agreements with US retailers and payors as well as exclusive distribution agreements with minimum commitments, across additional markets globally, including Mexico, South Korea, Panama with extension to Argentina, Colombia, Venezuela, and Costa Rica, Brazil, Kuwait, Saudi Arabia, Croatia, Poland, Serbia, and Bosnia and Herzegovina, Turkey and Israel with a strong pipeline of new agreements for the remainder of 2026.

Lead FDA-track program targets a serious pediatric disease with no FDA-approved treatment and could qualify for a transferable priority review voucher. Highlights Two FDA designation applications submitted for creatine monohydrate in guanidinoacetate methyltransferase (GAMT) deficiency: Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD). First-in-class product opportunity. No FDA-approved treatment exists for GAMT deficiency. Families measure and mix high-dose creatine powder by hand, several times a day, for life. 3D printing for children. Weight-banded, taste-masked, 3D-printed dissolvable tablets give each child a precise dose that can change as they grow. Voucher potential. If approved, the program could earn a rare pediatric disease priority review voucher (PRV). Recent vouchers have sold for US$150-200 million. Upcoming milestones. FDA decisions on both designations and a pre-IND meeting with the FDA are expected in Q1 2027.

Financial Results to Be Released Before Market Open