Product Updates at a Glance

Hemophilia A News

Sanofi and Sobi announced on 3/9/22 that their Phase 3 XTEND-1 BIVV001, Efanesoctocog alfa, a once-weekly treatment for Hemophilia A met its primary endpoints to provide statistically significant and meaningful reduction in annualized bleed rates compared to standard FVIII prophylactic treatment.

Efanesoctocog alfa is designed to provide near normal factor activity levels for the majority of the week.

https://www.sanofi.com/en/media-room/press-releas es/2022/2022-03-09-06-00-00-2399557

Hemophilia Gene Therapy News

BioMarin announced positive results on 2/4/22 at the 15th annual Virtual Congress of the European Association for Haemophilia and Allies Disorders (EAHAD) from their 2-year analysis for their Phase 3 GENEr8-1 Roctavian (valoctocogene roxaparvovec) therapy.

They reported that nearly all participants did not require standard preventive therapies, and that 80% of the participants were bleed free at the end of the 2-year period.

https://www.prnewswire.com/news-releases/biomarin-announcesoral-presentation-of-2-year-analysis-of-largest-phase-3-genetherapy-study-in-adults-with-severe-hemophilia-a-at-15th-annual-congress-of-european-association-for-haemophilia-and-allieddisorders-eahad-2-4-febr-301475471.html

Hemophilia B Gene Therapy News

CSL Behring announced on 2/4/22 at the 15th annual Virtual Congress of the European Association for Haemophilia and Allies Disorders (EAHAD) from their Phase 3 HOPE-B study on EtranaDez (etranacogene dezaparvovec) formerly known as AMT061, demonstrates durable and sustained therapeutic effects https://www.prnewswire.com/news-releases/finalanalysis-of-pivotal-hope-b-study-demonstrates-durableand-sustained-therapeutic-effect-of-etranacogenedezaparvovec-gene-therapy-in-hemophilia-b--datapresented-at-eahad-2022-301475671.html

Participants yielded stable FIX activity 18-months post treatment with 98% of study participants able to discontinue use of prophylaxis.

VonWillebrand News

The FDA granted approval on 1/31/22 for the first and only routine prophylactic treatment with VONVENDI for adult patients (18 and over) living with Severe Type 3 vonWillebrand Disease (vWD.) VONVENDI is approved for use for on-demand and perioperative bleed management for adults with vWD.

VonWillebrand Disease is considered to be the most common bleeding disorder with approximately 3 million (1 in every 100) individuals affected in the United States.

https://www.businesswire.com/news/home/20220131005214/en/ FDA-Approves-Prophylactic-Treatment-with-VONVENDI®-vonWillebrand-Factor-Recombinant-for-Adult-Patients-Living-withSevere-Type-3-von-Willebrand-Disease-VWD