Word from Washington: May 2023

person holding folder in front of Capitol

Lawmakers in May devoted close attention to the role that pharmacy benefits managers (PBMs) play in our health care system.  PBMs are middlemen that create and administer drug formularies on behalf of health insurers, self-insured employers, Medicaid, and other payers. PBMs choose which drugs a plan will cover, negotiate drug prices on behalf of payers, […]

Pfizer Announces Positive Results from Hemophilia A and B Trial

Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects. Marstacimab, a novel, investigational anti-tissue factor pathway inhibitor (anti-TFPI) being studied for the treatment of hemophilia A or B for people without inhibitors to Factor VIII (FVIII) or Factor IX […]

FDA Finalizes Blood Donor Eligibility Guidance

blood donation

On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based questions. Consistent with FDA’s January 2023 proposed guidance, the final guidance eliminates time-based donation deferrals and screening questions specific to men who have sex with men (MSM) and women who have […]

FDA issues response to Novo Nordisk’s request for concizumab approval

Recently Novo Nordisk received a Complete Response Letter from the Food and Drug Administration for concizumab. Concizumab was submitted for regulatory approval in the U.S. for the treatment of hemophilia A and B with inhibitors in August 2022. In the letter, the FDA requested additional information related to the monitoring and dosing of patients to […]

The HELP Copays Act moves forward – now in the Senate as well as in the House 

building facade

Protections for people who rely on copay assistance have been a top priority for HFA and many other patient groups for several years. That effort took a big step forward in April with the introduction of a first-ever Senate version (S. 1375) of the HELP Copays Act.   Sponsored by Sens. Kaine, Marshall, Markey, and Ernst, […]

HFA Welcomes Dan Kelsey as Chief Executive Officer

Dan Kelsey Headshot

Experienced executive brings outcome-driven leadership to the national nonprofit patient advocacy group.  WASHINGTON, DC, April 12, 2023 – The Board of Directors of Hemophilia Federation of America (HFA) unanimously selected Dan Kelsey as HFA’s new Chief Executive Officer (CEO) on April 7, 2023. Dan will join the HFA team on Wednesday, April 12 and attend […]

Sanofi: two published studies highlight fitusiran’s potential to address unmet needs

microscope in lab

Two fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia Paris – April 4, 2023 – Two studies, published in The Lancet and The Lancet Haematology, evaluating the efficacy and safety of fitusiran, an investigational siRNA therapy for the prophylactic treatment of adults and adolescents with hemophilia A or B, reinforce the potential […]

State of the States: Spring 2023

person holding folder in front of Capitol

States can resume Medicaid terminations in April, but most are holding back  The U.S. Department of Health and Human Services (HHS) has notified state Medicaid programs that the COVID-19 public health emergency (PHE) will formally end on May 11, 2023.  The PHE has been continuously renewed in 90-day increments since first declared by HHS in […]

The Importance of Copay Assistance Protections: Awareness Grows 

building facade

For over three decades, March has been designated “Bleeding Disorders Awareness Month.” HFA and member organizations across the country mark the occasion with monthlong education and advocacy activities – social media outreach, state and local proclamations and resolutions, Hill Days, and more.  In March 2023, as in past years, HFA was pleased to take part […]

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 […]