FDA blocks much anticipated BioMarin hemophilia gene therapy

BioMarin Pharmaceutical shares plummeted by a third after U.S. regulators rejected their potentially game-changing gene therapy for hemophilia A patients

FAIRLESS HILLS, PA – Investors fled drug developer BioMarin in drowsiness on Wednesday, driving shares up a third after U.S. regulators rejected the company’s potentially game-changing hemophilia. A gene therapy about worries that it might not really be a one-and-a-half life treatment.

The U.S. Food and Drug Administration’s rejection late Tuesday means the San Rafael, California-based company must complete an ongoing late-stage patient study, likely delaying possible approval until late 2022.

The infusion therapy, called Roctavian, could have freed hemophilia A patients from frequent, extremely expensive infusions of a blood clotting therapy to prevent dangerous internal bleeding. It was highly anticipated by doctors, patients and investors.

In a statement, BioMarin said the company and the FDA had previously agreed on how much data for patient testing the agency needed to evaluate the therapy, but in its waiver letter, the FDA first recommended Biomarin complete the end-of-stage study and two years of providing follow-up data on the safety and effectiveness of therapy in preventing internal bleeding for all study participants.

The company added that FDA conclusions concluded between the results of a small, early-stage study and interim data from the late-stage study left unclear how long the effect of the therapy would last.

Roctavian was intended to relieve patients with severe hemophilia A from 100 to 150 Factor VIII IV infusions per year to reduce painful, spontaneous bleeding in joints and muscles, which could cause permanent damage.

Also known as valoctocogenic roxaparvovec or valrox for short, it would have been the first gene therapy approved in the US for any type of hemophilia. This is a rare, genetic bleeding disease in which humans do not have enough of a clotting protein called Factor VIII due to a mutation in the gene responsible for producing it. They suffer spontaneous internal bleeding several times. About 1 in 10,000 people, mostly men, have hemophilia A, including about 20,000 in the US About half have serious illness.

The gene therapy works by using an inactivated virus, created in a laboratory, to deliver an inactive liver via a single IV infusion intended to enable the body to produce FVIII on its own.

Questions about whether it would work for a lifetime or just a few years came amid rumors that Biomarin could set a price tag of up to $ 3 million per patient. That would top the price for the most expensive therapy ever approved by the FDA, Swiss drugmaker Novartis AG’s gene therapy for spinal atrophy, Zolgensma, which was launched in the spring of 2019 with a price tag of $ 2.125 million per patient.

Biomarin has estimated the cost of living current treatments to prevent bleeding at about $ 25 million, arguing that its gene therapy would save far more than its cost.

Several other drugmakers are developing gene therapies for hemophilia A, but are further behind in testing: partners Pfizer Inc. and Sangamo Therapeutics, Spark Therapeutics and Generation Bio.

SVB Leerink analyst Joseph Schwartz on Wednesday closed his 12-month price target for BioMarin’s share from $ 140 to $ 113 per share. He called the FDA ruling a “major negative surprise”, but added, “We would not sell the stock here, as disappointing as it is.”

Schwartz notes that after BioMarin revealed disappointing data from an interim analysis of the ongoing study after the late phase, company shares dropped to $ 63. Schwartz believes that BioMarin’s other assets are worth a share price of $ 88.

The company currently sells six drugs in the US, all for rare genetic or enzyme disorders. It posted a profit of $ 52 million on revenue of $ 932 million in the first six months of 2020.

In mid-afternoon trading, shares of Biomarin Pharmaceutical Inc. $ 42.62, or 36%, to $ 75.92. However, the trading volume was about 19 times the normal number of BioMarin shares in a day.


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