Pfizer has announced a successful large late-stage trial in experimental gene therapy for Hemophilia - a rare, inherited genetic disorder that prevents the body from clotting blood properly. The condition causes people to bleed for longer after injuries, bruise easily, and have an increased risk of bleeding into their joints or brain.

This development, according to experts, is slated to pave the way for a potential approval for a treatment for hemophilia A, which could become the company’s second gene therapy to enter the US market after Beqvez, which was cleared earlier this year.

According to the biopharmaceutical company headquartered in New York, they have been co-developing the therapy along with Sangamo Therapeutics to reduce patients’ bleeds for at least 15 months. Even though the data may be enough to muster approval, they are unlikely to quell doubts about whether the treatment can provide a cure - as hoped - for a meaningful option for most patients in the US and Europe with the rare blood disorder. The company said the drug also performed better than the current standard treatment for the disease, which is routine infusions that replace the Factor VIII protein.

Even though the exact number of people living with hemophilia in the US is not known, the CDC estimated that as many as 33,000 males in the country are living with the disorder. India is home to the second largest population of patients living with Hemophilia, with an estimated 1,36,000 individuals grappling with hemophilia A. However, only around 21,000 are registered at present.

Hemophilia A, also known as classical hemophilia, is a hereditary hemorrhagic disorder resulting from a congenital deficit of factor VIII that manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. It is a life-long disease that increases the risk of spontaneous bleeding and severe bleeding after surgery. According to statistics, the condition occurs in roughly 25 in every 100,000 male births worldwide.

Doctors say people can be born with mild, moderate, or severe forms of hemophilia A and develop spontaneous bleeding into their joints which is extremely painful, affecting their ability to get around. Bleeding in other parts of your body is also possible.

According to studies, hemophilia B is less serious than hemophilia A but is still a serious medical condition. Doctors say those who have B may have fewer problems with excessive bleeding. A few differences between the two, include:

• People who have hemophilia B have less hemarthrosis - bleeding into their joints, and less joint damage from that bleeding into their joints.
• People with hemophilia B have fewer episodes of spontaneous bleeding
• Sometimes, people treated for hemophilia develop problems with antibodies that interfere with treatment. Studies show people who have hemophilia B are less likely to develop these issues.

Pfizer says further trials in the offing

“For people living with hemophilia A, the physical and emotional impact of needing to prevent and treat bleeding episodes through frequent IV infusions or injections cannot be underestimated,” said Dr. Andrew Leavitt, the lead investigator of the trial, in a statement.

Pfizer said the study is ongoing and it will present additional data at upcoming medical meetings.

According to the company, if approved, their therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which has had a slow rollout since it won approval in the US last year, raising questions about how many patients would take Pfizer’s drug if it enters the market. News reports say BioMarin is also considering whether to divest its hemophilia A therapy, which costs $2.9 million.