Amid the delay in approval from USFDA, BioMarin’s first-of-its-kind hemophilia gene therapy has now got conditional approval in Europe. According to reports, the European Medicines Agency has recently recommended granting conditional approval to BioMarin’s Roctavian, also known as valoctocogene roxaparvovec, to treat patients with severe hemophilia A who do not have factor VIII inhibitors, the European regulator said last week. Moreover, the patients should also test negative for antibodies to the adeno-associated virus serotype 5, which serves as the vector for gene therapy.
The positive opinion sets Roctavian up for an official European Commission approval in the third quarter, BioMarin said as per reports. Reportedly, this move will make Roctavian the first gene therapy available in Europe for hemophilia A.
Meanwhile, the company expects its first rollout in Germany with a reimbursment agreement in place right after a potential approval, BioMarin Chief Commerical Officer Jeff Ajer told investors during a conference call Friday.
This comes at a time when BioMarin has yet to refile Roctavian with the FDA. Last month, the company postponed plans to resubmit an FDA application to September, saying the U.S. agency has requested additional information. In 2020, the FDA had rejected Roctavian, demanding longer, two-year annualized bleeding rate data from a phase 3 clinical trial. BioMarin shared those data in January and at that time laid out a plan to knock on the FDA’s doors again in the second quarter.
Now the positive EMA opinion bodes well for Roctavian in the U.S., SVB Securities analyst said in a Friday note.
As the EMA noted, currently available hemophilia A treatments require lifelong repeated injections. Roche’s Hemlibra, a leading antibody-drug, can be taken up to once every four weeks.
“Therefore, there is an unmet medical need for new therapeutic approaches that might free patients from frequent injections,” the EMA said in a statement.
According to the Pharma firm, Roctavian uses AAV5 to carry functional factor VIII genes into the liver cells to replenish the missing blood-clotting protein. Additionally, BioMarin is proposing Roctavian as a one-time therapy, but questions remain about the drug’s long-term efficacy.
In Roctavian’s phase 3 trial in 134 patients, the average annualized bleeding rate fell 85% from its baseline to less than one episode in the second year after treatment. BioMarin also submitted five-year data from a small early-phase trial, which the EMA said supported its treatment effect.
Nevertheless, the EMA said it’s unknown how long the effect from a single infusion will last in a patient. “Longer-term follow-up tests may be required to verify a continued safe and effective response to the medicine,” the EMA said of Roctavian.
Once Roctavian is officially approved, patients will be monitored for 15 years to ensure the long-term efficacy and safety of this gene therapy, the agency added.