FDA Blocks Much-Anticipated BioMarin Hemophilia Gene Therapy

August 20, 2020by Linda A. Johnson, AP Medical Writer
The U.S. Food and Drug Administration building behind FDA logos at a bus stop on the agency's campus in Silver Spring, Md. (AP Photo/Jacquelyn Martin)

FAIRLESS HILLS, PA (AP) — Investors fled drug developer BioMarin in droves on Wednesday, driving shares down by a third after U.S. regulators rejected the company’s potentially game-changing hemophilia A gene therapy over concerns it might not really be a one-and-done lifetime treatment.

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

The infused 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 had been highly anticipated by doctors, patients and investors.

In a statement, BioMarin said the company and the FDA previously agreed on how much patient testing data the agency required to review the therapy, but in its rejection letter the FDA for the first time recommended Biomarin finish the late-stage study and provide two years of follow-up data on the therapy’s safety and efficacy in preventing internal bleeding for all study participants.

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

Roctavian was meant to free patients with severe hemophilia A from 100 to 150 IV infusions of Factor VIII per year to prevent or at least reduce painful, spontaneous bleeding into joints and muscles, which can cause permanent damage to them.

Also known as valoctocogene roxaparvovec or valrox for short, it would have been the first gene therapy approved in the U.S. for any type of hemophilia. That’s a rare, genetic bleeding disorder in which people don’t have enough of a clotting protein called Factor VIII due to a mutation in the gene responsible for producing it. They repeatedly suffer spontaneous internal bleeding. About 1 in 10,000 people, mostly males, have hemophilia A, including about 20,000 in the U.S. About half have severe disease.

The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant 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 might set a price tag as high as $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 muscular atrophy, Zolgensma, which was launched in spring 2019 with a $2.125 million price tag per patient.

Biomarin has estimated the lifetime cost of current treatments to prevent bleeding at about $25 million, arguing 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 slashed his 12-month price target for BioMarin’s stock 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 this is.”

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

The company currently sells six medicines in the U.S., 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, Biomarin Pharmaceutical Inc. shares plunged $42.62, or 36%, to $75.92. Trading volume by then was about 19 times the usual number of BioMarin shares traded in a day.

In The News

Health

Voting

Regulation

After 9 Years and $10M, Georgia Spaceport Nears FAA Approval
In The States
After 9 Years and $10M, Georgia Spaceport Nears FAA Approval

SAVANNAH, Ga. (AP) — After nine years of planning and $10 million invested by local taxpayers, county officials in Georgia's coastal southeast corner came a big step closer Thursday to winning federal approval of a project engineered to literally inject the local economy with rocket fuel.... Read More

Biden's Budget Seeks Bigger Investments And Expanded Authority For FDA
Federal Budget
Biden's Budget Seeks Bigger Investments And Expanded Authority For FDA
June 2, 2021
by Daniel Mollenkamp

WASHINGTON- The Biden Administration's 2022 budget, released at the end of last week, seeks significant spending increases for the U.S. Food and Drug Administration.  The fiscal year 2022 budget request sets out $6.5 billion for the FDA, an increase of almost 8% ($477 million) over last... Read More

CEOs Say Regulatory Reform Key to Climate, Infrastructure Plan Success
Regulation
CEOs Say Regulatory Reform Key to Climate, Infrastructure Plan Success
May 19, 2021
by TWN Staff

If the White House and Democrats in Congress truly want to achieve President Joe Biden's economic goals, they should not overlook the need to modernize and improve the nation's cumbersome regulatory system, according to a new report from The Business Roundtable, a trade association of company... Read More

FDA Expected to OK Pfizer Vaccine for Teens Within Week
Health
FDA Expected to OK Pfizer Vaccine for Teens Within Week

WASHINGTON (AP) — The U.S. Food and Drug Administration is expected to authorize Pfizer's COVID-19 vaccine for youngsters ages 12 to 15 by next week, according to a federal official and a person familiar with the process, setting up shots for many before the beginning of... Read More

Supreme Court Upholds FCC Bid to Loosen Media Ownership Rules
Supreme Court
Supreme Court Upholds FCC Bid to Loosen Media Ownership Rules
April 1, 2021
by Dan McCue

WASHINGTON - The Supreme Court on Thursday upheld a decision by the Federal Communication Commission to relax media ownership rules. The unanimous ruling, written by Justice Brett Kavanaugh, is a major win for broadcasters. The petitioners in the case, the Prometheus Radio Project, were seeking a... Read More

Social Media Platforms’ Self-Regulation Era is Over
Social Media
Social Media Platforms’ Self-Regulation Era is Over
March 25, 2021
by Victoria Turner

WASHINGTON - Rep. Cathy McMorris Rogers, R-Wash., said at a congressional hearing today that as a parent of three school-aged children, Facebook, YouTube and Twitter are her “biggest fear.”  After spikes in teenage suicides in her community, everyone she reached out to “[raised] the alarm about... Read More

News From The Well
scroll top