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FDA Approves First Gene Therapy For Severe Hemophilia A

The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.

The treatment is not cheap: Roctavian will cost $2.9 million for a single infusion, the Associated Press reported.

"Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding," Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in an agency news release. "Today's approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy."

For patients, this means a potential reduction in uncontrolled bleeding. Left untreated, severe hemophilia A can cause life-threatening bleeding into vital organs, including the kidneys and brain.

Drug maker BioMarin Pharmaceutical Inc. Told the AP that the drug's astronomical price tag reflects "the possibility of freedom from years" of infusions with other medications. Those infusions typically run about $800,000 each year for patients, the AP said.

Hemophilia A is caused by a mutation on a gene which produces a protein that enables blood to clot. This protein is called Factor VIII. The condition primarily affects males. How frequent and severe bleeding episodes are depends on the amount of FVIII protein a person produces. In about 60% of cases, people with severe hemophilia A have less than 1% of FVIII in their blood.

Before now, treatment has involved FVIII replacement therapy or an antibody-based medication, the FDA said.

Roctavian is a viral vector that carries the gene for Factor VIII. It reduces the risk of uncontrolled bleeding by delivering the gene into the liver, to increase blood levels of FVIII.

The approval was based on a multinational study in adult men ages 18 to 70 who had previously had FVIII replacement therapy determined the drug's safety and effectiveness. A total of 112 patients were followed for at least three years.

Those treated in the study had far fewer bleeding incidents. Their mean annualized bleeding rate dropped from 5.4 times each year to 2.6. Most of those who received Roctavian also took corticosteroids to suppress their immune system. The FDA noted that treatment response may decrease over time.

Adverse reactions seen in the study included mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain and infusion-related reactions.

The treatment is not without risks. The FDA recommends close monitoring for infusion-related reactions and elevated liver enzymes. An increase in FVIII activity could potentially increase the risk of blood clots, the FDA noted. The product also carries a potential cancer risk.

More information: The National Hemophilia Association has more on hemophilia A.

Citation: FDA approves first gene therapy for severe hemophilia A (2023, July 3) retrieved 3 July 2023 from https://medicalxpress.Com/news/2023-07-fda-gene-therapy-severe-hemophilia.Html

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US Regulator Approves First Ever Gene Therapy For People With Hemophilia A

A life-changing treatment for people with hemophilia A, which was developed at UCL and licensed by UCLB to BioMarin Pharmaceuticals Inc., has been approved by the United States' Food and Drug Administration (FDA).

Roctavian (valoctocogene roxaparvovec) has become the first ever gene therapy for adults with severe hemophilia A to be approved by the FDA, meaning it can be sold as a treatment for patients with the disease. It follows a similar approval in August 2022 by the European Commission.

Hemophilia A is a rare genetic bleeding disorder, where a genetic mutation results in a deficiency of Factor VIII, a blood clotting protein normally produced by the body, which can mean bleeding is harder to control and is life-threatening in severe cases. Hemophilia A can seriously impact on quality of life because of the harm from bleeding, the need to manage the risk caused by physical activity, and the limitations of previous treatments.

Currently, hemophilia A requires ongoing treatment throughout patients' lives, with regular injections to replace the missing Factor VIII.

Roctavian is ground-breaking because it is a one-time, long-term treatment, using a gene therapy approach to treat the disease and enable people with hemophilia A to increase their levels of Factor VIII. The active Factor VIII gene is delivered by an engineered adeno-associated virus to deliver genetic material into cells. The gene is then expressed in the liver, which produces the Factor VIII protein, improving the ability of the body to control bleeding without regular injections.

The technology was developed by Professor Amit Nathwani (UCL Cancer Institute and Institute of Immunity & Transplantation) and his team at UCL and St. Jude Children's Research Hospital, U.S. It was licensed by UCLB, the commercialization company for UCL, to BioMarin Pharmaceuticals Inc. In 2013.

Professor Nathwani said, "It is humbling to be involved in the creation of the first gene therapy for patients with severe hemophilia A. Current treatment is effective but highly demanding requiring regular lifelong injections. Most severe hemophilia A patients suffer recurrent break-through bleeding into joints despite treatment that ultimately leads to disability and chronic pain. The approval of Roctavian, is an important and long-awaited advance that addresses the genetic cause of the condition. Roctavian is a one-time gene therapy that offers patients long term protection from bleeds and avoids the burden of regular infusions."

Biomarin's GENEr-8, the longest global Phase 3 study to date for any gene therapy in hemophilia, was key to achieving the approval of Roctavian by the FDA. It found that the therapy was effective in reducing the rate of bleeding in a cohort of 134 patients for at least three years.

Professor Geraint Rees, UCL Vice-Provost (Research, Innovation and Global Engagement) said, "Creating the first long-term treatment for severe hemophilia A will change lives, and proving its effectiveness and achieving FDA approval is a major achievement. It is a prime example of how our approach to industry-academic engagement is directly benefitting society."

UCL is a recognized leader in transformative gene therapies. The success of Roctavian follows a number of recent milestones for UCLB's gene therapy spinouts, including Freeline's first patient dosed with a novel gene therapy candidate for Gaucher Disease, and the use of spinout Orchard Therapeutic's Libmeldy by the NHS to treat children with metachromatic leukodystrophy, an otherwise fatal disorder.

Richard Fagan, Director of BioPharm at UCLB, said, "Our partnership with BioMarin has spanned over a decade. Since licensing the technology to BioMarin, the company has taken forward Roctavian's pioneering clinical development, and undertaken some of the most extensive clinical trials for a gene therapy in the world. The successful FDA approval is a significant achievement for UCL and UCLB and ultimately Hemophilia patients."

Provided by University College London

Citation: US regulator approves first ever gene therapy for people with hemophilia A (2023, July 3) retrieved 3 July 2023 from https://sciencex.Com/wire-news/449859581/us-regulator-approves-first-ever-gene-therapy-for-people-with-he.Html

BioMarin's Delayed Roctavian Scores FDA Nod As First Hemophilia A Gene Therapy In US

The FDA approved BioMarin Pharmaceutical Inc's (NASDAQ:BMRN) Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5.

The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was approved by the European Medicines Agency in August 2022.

In March, the FDA said it needed more time to review a three-year analysis from BioMarin's ongoing Phase 3 GENEr8-1 study of Hemophilia A gene therapy, which BioMarin submitted earlier this year.

Hemophilia A is a genetic condition caused by a mutation in the gene responsible for producing a protein called FVIII, which is necessary for blood clotting.

Roctavian is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.

Roctavian is manufactured at a BioMarin-owned facility in Novato, California.

BioMarin will continue to monitor the long-term effects of treatment with an extension study that will follow all clinical trial participants for up to 15 years, as well as post-approval studies to follow those dosed in a real-world setting for 15 years or more.

It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive Roctavian with this initial approval.

William Blair writes that the impressive early launch of Voxzogo, the company's largest approved product to date, and U.S. And EU approvals of Roctavian represent major additional growth drivers.

The analyst sees strong potential for BioMarin to double its top line in the coming years.

Noting the company's deep rare-disease pipeline, with a promising growth profile, the analyst views BioMarin as a core holding in the sector and rates BioMarin at Outperform.

Truist Securities analyst Robyn Karnauskas maintained Biomarin Pharmaceutical with a Buy and raised the price target from $125 to $140.

Wedbush analyst Andreas Argyrides maintained Biomarin with a Neutral and raised the price target from $69 to $73.

Price Action: BMRN shares are up 3.17% at $91.75 premarket on the last check Friday.

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