Roche’s (RHHBY) Hemlibra is used for the prevention and reduction of the frequency of bleeding episodes in individuals with hemophilia A with factor VIII inhibitors.
HAVEN clinical trial program
In December 2017, Roche presented the results from the phase three HAVEN 4 trial. The trial evaluated the safety and efficacy of a Hemlibra dose once every four weeks in adults and adolescents with hemophilia A with or without factor VIII inhibitors.
In December 2017, Roche also presented the long-term results of the pivotal HAVEN 1 and HAVEN 2 trials at the 59th American Society of Hematology Annual Meeting.
In the phase three HAVEN 2 trial, 94.7% of children with hemophilia A with factor VIII inhibitors who were on Hemlibra prophylaxis showed zero treated bleeds. The intra-patient analysis relating to the effects of diverse therapies in the same children demonstrated a 99% reduction in treated bleeds with Hemlibra prophylaxis versus prior treatment with bypassing agents (or BPA) either as prophylaxis or on request.
Hemlibra prophylaxis also demonstrated significant progress in health-related quality of life and features of caregiver burden, calculated by the Haemo-QoL-SF (hemophilia-specific quality of life short form) and Inhib-QoL (adapted health-related quality of life in hemophilia patients with inhibitors) questionnaires compared to prior BPA prophylaxis.
After ten months, HAVEN 1 trial results indicated that intra-patient analysis demonstrated that patients on Hemlibra prophylaxis showed an 88% reduction in treated bleeds compared to previous BPA prophylaxis. The HAVEN 1 trial also demonstrated a 95% decrease in treated bleeds in patients who received Hemlibra prophylaxis compared to previous on-demand BPA therapy.
In the hemophilia drugs market, Roche’s peers include Bioverativv (BIVV), Shire (SHPG), Novo Nordisk (NVO), and Bayer (BAYZF). In 3Q17, Shire, Novo Nordisk, and Bayer reported revenues of $3.7 billion, $4.2 billion, and 8.0 billion euros, respectively. The Vanguard FTSE Developed Markets ETF (VEA) invests ~2.1% of its total portfolio holdings in Roche.