Thursday, February 2, 2023
ADVT 
Health

FDA blocks much-anticipated BioMarin hemophilia gene therapy

Darpan News Desk The Canadian Press, 19 Aug, 2020 08:31 PM
  • FDA blocks much-anticipated BioMarin hemophilia gene therapy

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 rumours 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.

MORE Health ARTICLES

'We're the pioneers': Canadian COVID-19 survivors share their stories

'We're the pioneers': Canadian COVID-19 survivors share their stories

We all know we should be afraid of it, but relatively few Canadians have experienced COVID-19 themselves.

 

The effort to contain the pandemic has wrought seismic disruptions to daily life on an individual and global scale. But even as the number of cases in Canada climbs, it's hard to comprehend the contagion based on a list of symptoms and warnings that it's fatal.

'We're the pioneers': Canadian COVID-19 survivors share their stories

Is COVID-19 baby boom a myth? How relationships might be tested during the pandemic

Is COVID-19 baby boom a myth? How relationships might be tested during the pandemic

CALGARY — As Canadians face the possibility of being alone with their significant other for the foreseeable future, some say marital discord is more likely than the kind of intimacy that would lead to a baby boom nine months from now.

Is COVID-19 baby boom a myth? How relationships might be tested during the pandemic

Fertility startup promises to measure women's fertility through at-home AMH tests

Fertility startup promises to measure women's fertility through at-home AMH tests

Caitlin Blaney always dreamed of being a mom. But thoughts of having children got put aside while she pursued her education to become a clinical psychologist. 

Fertility startup promises to measure women's fertility through at-home AMH tests

Medications Put Older People At Severe COVID-19 Risk

The researchers recommended future case-control studies in patients with COVID-19 infections to further confirm chronic therapy with ACEIs or ARBs may raise the risk for severe outcomes.

 

 

Medications Put Older People At Severe COVID-19 Risk

Babies Frequently Exposed To Cleaning Products At Higher Risk Of Asthma: Study

Babies Frequently Exposed To Cleaning Products At Higher Risk Of Asthma: Study

The CHILD Cohort Study found young infants living in homes where cleaning products were used frequently were more likely to develop childhood wheeze and asthma by age three.

Babies Frequently Exposed To Cleaning Products At Higher Risk Of Asthma: Study

E-Cigarettes More Harmful To Heart Than Tobacco: Study

Researchers have found that electronic nicotine delivery systems, such as e-cigarettes might be just as harmful to the heart, than traditional cigarettes.

 

 

E-Cigarettes More Harmful To Heart Than Tobacco: Study