BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023

SAN RAFAEL, Calif., March 6, 2023 — BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults […]

The Complexity of Unwinding Medicaid

The long-heralded Medicaid “unwinding” has begun. As previously reported, state Medicaid programs have, since the start of the COVID-19 pandemic, operated under a law that prohibited them from disenrolling Medicaid enrollees for the duration of the public health emergency (PHE). Congress changed that requirement in December 2022, delinking the Medicaid continuous enrollment obligation from the PHE. […]

FDA Approves Sanofi’s Once-Weekly ALTUVIIIO™


Source: Sanofi Press Release Sanofi announces that the U.S. Food and Drug Administration approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-vWF-XTEN Fusion Protein-ehtl], previously known as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.  Sanofi states that with ALTUVIIIO, they are looking to raise the bar for people with […]

Dear Addy: Project CALLS

medical supplies

Dear Addy,  I recently attended an event and learned about submitting issues with my insurance to HFA’s Project CALLS. I have an insurance issue, however, I still have some questions about how CALLS works and what happens when I submit an issue. Can you explain the process?  Signed,  CALLS Quandary   Dear Quandary,  You are not […]

Finding Strength Through Diversity

group of young adults in front of Capitol

This Black History Month, HFA asks our community to think back on the family members they’ve loved and the moments they created. We ask our community to think on the diversity of our community, the stories we’ve shared, and the people of color who helped make the bleeding disorder community as strong as it is […]

Word from Washington: January 2023

person holding folder in front of Capitol

HFA reports on a particularly concerning insurance problem unfolding in Tennessee, and how HFA is responding; news on Medicaid eligibility reviews; changes in blood donation policy; and much more.

FDA Proposes Blood Donation Changes

red blood cells

On Friday, January 27, 2023, the U.S. Food and Drug Administration announced proposed changes to national blood and plasma donation policies. The draft FDA guidance, if finalized, would eliminate time-based donation deferrals for men who have sex with men. Instead, revised donor history questionnaires would ask all donors (regardless of gender) to answer individual risk-based […]

WFH Gene Therapy Registry: now live!

The World Federation of Hemophilia (WFH) has developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR). The launch of the WFH GTR coincides with the first approvals of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

HFA & NHF Respond to New Blue Cross Blue Shield of Tennessee Formularies

medical supplies

On January 1st, Blue Cross Blue Shield of Tennessee (BCBST) implemented formularies for their health plans (individual, small group, and large group) that remove 17+ bleeding disorders treatments from coverage. These exclusions threaten the health of potentially hundreds of Tennesseans who live with inherited blood or bleeding disorders. Patients who have been stable on their […]

Dear Addy: Pharmacy Choice

medical supplies

A community member writes in about their struggles with a friend who is pressuring them to switch to the specialty pharmacy that they work for. Addy share tips with writer as how to try to both keep their friendship and their pharmacy.