FDA approves world’s most expensive drug

The US Food and Drug Administration (FDA) has approved CSL Behring's hemophilia B gene therapy, a single-infusion treatment with a list price of $3.5 million, making it the most expensive drug in the world.

CSL Behring's approval of Hemgenix "provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those experiencing a high disease burden associated with this form of hemophilia," he said. Peter Marks, director of the FDA's Center for Biologicals Evaluation and Research.

Hemgenix is ​​used to treat patients with haemophilia B who are currently using prophylactic factor IX therapy or who have severe spontaneous bleeding episodes or have had life-threatening bleeding. Based on clinical studies, the infusion reduced annual bleeds, and "94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free," the company said, opening up the possibility for the drug to eliminate factor IX. need for routine lifelong treatment in adult patients.

Resulting from a single gene defect, haemophilia B is a rare, lifelong bleeding disorder whose currently available treatment requires patients to undergo rigorous lifelong prophylactic infusions of factor IX. While effective, patients are prone to spontaneous bleeding episodes, while Hemgenix allows people to "make their own factor IX," according to CSL Behring.

Hemgenix treats patients at the genome level, with an engineered virus that carries the gene expressed in the liver to produce clotting factor IX. Gene therapies are believed to significantly improve medical conditions by addressing the underlying causes.

“Even if the price is a little higher than expected, I think it has a chance of success because 1) existing drugs are also very expensive and 2) patients with haemophilia live in constant fear of bleeding,” he told Bloomberg Brad Loncar, chief executive officer of Loncar Investments. “A gene therapy product will be attractive to some.”

The one-time treatment from Hemgenix may allow people to avoid regular infusions from current treatment providers, Biogen and Pfizer. The list price isn't necessarily what patients pay for the drug, thankfully, because there will be government subsidies.

Hemophilia is a bleeding disorder characterized by the body's inability to clot blood properly due to a lack of sufficient clotting proteins or clotting factors. It is almost always a genetic disease, which is of concern when the bleeding occurs internally and leads to life-threatening organ and tissue damage. Symptoms include excessive bleeding from cuts, blood in the urine, and bleeding in the brain. Many people are born with this disorder, and the most common type is haemophilia A with low factor VIII, followed by haemophilia B with low factor IX. Acquired hemophilia can be caused by autoimmune conditions and adverse drug reactions.

According to the Mayo Clinic, hemophilia almost always occurs in boys and is passed from mother to child through one of the mother's genes. The Centers for Disease Control and Prevention (CDC) state that hemophilia occurs in about 1 in 5,000 male births. A known case of hemophilia was that of the son of Tsar Nicholas II, Alexei Romanov, whose disease influenced Russian history through Rasputin's healing intervention. The FDA approved Hemgenix following results from a multinational, open-label, single-arm, Phase III HOPE-B clinical study evaluating the safety and efficacy of the treatment.

Based on a study of 54 participants, Hemgenix increased factor IX activity levels and reduced the need for routine replacement prophylaxis, while there was a 54% reduction in the annual bleeding rate from baseline. The gene therapy allowed the patients to produce an average factor IX activity of 39 percent at six months and 36.7 percent at 24 months after infusion, CSL Behring said. After treatment, 51 of the 54 participants discontinued prophylactic use and remained free from previous continuous prophylactic therapies.

The most common adverse effects following Hemgenix gene therapy were elevated liver enzymes, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling sick.

So anyway it's a big step forward for medicine: a bit expensive, but life is priceless.

---

---

The article FDA approves world's most expensive drug comes from Economic Scenarios .

---

This is a machine translation of a post published on Scenari Economici at the URL https://scenarieconomici.it/la-fda-approva-il-farmaco-piu-costoso-al-mondo/ on Sat, 26 Nov 2022 16:00:04 +0000.