Approval, check. Now, can BioMarin make a commercial success of its hemophilia A gene therapy in Europe?

Despite regulatory hang-ups in the U.S., BioMarin’s Roctavian has passed muster in Europe as the bloc’s first hemophilia A gene therapy.

Wednesday, the European Commission gave Roctavian the all-clear, granting conditional marketing authorization to the med in adults with severe hemophilia A who don’t have factor VIII inhibitors, BioMarin said in a release.

In tandem with the green light, Roctavian boasts an orphan-drug designation in the EU, which comes with a coveted 10 years of market exclusivity.

Still, the California-based company will have its work cut out convincing overseas payers of the pricey drug's value.

BioMarin’s one-and-done gene therapy works by delivering a functional gene designed to help the body produce factor VIII by itself, without the need for continued hemophilia prophylaxis. People with hemophilia A suffer from a mutation in the gene responsible for producing factor VIII, which is a protein essential to blood clotting, BioMarin explained.

Roctavian represents one of two “transformational” launches for BioMarin, the other being achondroplasia treatment Voxzogo, CEO Jean-Jacques Bienaimé said last summer. Voxzogo has since hit the U.S. market following its FDA approval in November 2021.

Across the pond, BioMarin has yet to file Roctavian with the FDA. Back in June, the company delayed plans to resubmit its application until September. It explained that the FDA had requested additional information.

Still, BioMarin’s EU approval is nothing short of an “epic accomplishment,” Evercore ISI analysts wrote in a note to clients Thursday. They flagged the fact that short-acting factor replacement formed the backbone of hemophilia A care less than 10 years ago.

Roctavian’s net price in Europe will clock in at around 1.5 million euros, the Evercore team said, citing comments made by BioMarin on a recent conference call.

Across the bloc, the company is targeting some 3,200 patients based on Roctavian’s label, which also excludes those who test positive for antibodies to the adeno-associated virus serotype 5, which serves as the vector for the gene therapy.

That said, Roctavian could over time grow its label and cover more of the EU’s severe hemophilia A population of about 8,000, the analysts said.

To start, BioMarin will launch in Germany, where the company expects to begin commercial dosing in the fourth quarter. The company could be looking at about 600 to 700 eligible patients early on, “with some initial pent-up demand,” the Evercore team added.

From there, Roctavian will make its way to France, where it could pick up another 500 to 600 patients, before stopping over in Italy and Spain, then other EU countries, and, finally, the Middle East.

The Evercore team figures it’s reasonable to assume Roctavian could pull down sales of $21 million this year. Next year’s forecast of $260 million is “also doable,” the team noted.

With Roctavian, BioMarin will be attempting to succeed where bluebird bio's beta thalassemia gene therapy Zynteglo fell short. After bluebird and national reimbursement authorities in Europe reached a stalemate over fair gene therapy pricing, the company last summer said it would “wind down” its operations in Europe to focus on business in the U.S. Zynteglo launched in Europe at a price of $1.8 million.

As for Roctavian, the EU approved BioMarin's gene therapy based on several studies that included up to five years of patient follow-up.

Back in 2020, the FDA surprisingly turned down 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.

Nevertheless, BioMarin has cause to feel emboldened after Wednesday’s approval. Stateside, BioMarin confirmed it continues to target September for the resubmission of its Roctavian FDA application.

In addition to the standard six months of review time that follows the filing of a biologics license application, BioMarin expects the U.S. regulator may need “three additional months” thanks to the number of data readouts expected to surface during the review period.