Hemophilia

University of Pittsburgh’s Margaret Ragni, M.D., M.P.H., discussed fitusiran, the investigational small interfering RNA (siRNA) therapeutic, with Managed Healthcare Executive and also some of the possible limitations of gene therapy for hemophilia.

A yearlong study of the newly approved half-life extender showed no evidence of factor VIII inhibitors.

Promising results were reported in The Lancet and The Lancet Haematology.

The price tag of $2.9 million is typical of a new wave of gene therapies. The manufacturer, BioMarin Pharmaceutical, is offering arrangements that will link the price paid by payers to how well patients respond to the treatment.

Hemophilia A is seen as a male-only disease, but this study shows the extent to which female carriers also suffer from bleeding and joint problems related to reduced levels of clotting factors VIII.

The researchers concluded that delayed prophylaxis start with intermediate dose intensity of factor VIII prevents bleeds, but at a cost of more joint damaged and reduced health-related quality of life.

FDA has announced evidence-based changes that will allow more individuals to donate blood without increasing the risk of HIV infection.

How long the gene therapies for hemophilia will keep factor levels high is unclear, said the authors of a review in the Annual Review of Medicine. Patients also need to be counseled that they can receive only one adeno-associated viral (AAV) vector in their lifetime.

Nonexclusive licensing of discoveries made at academic institutions would work to lower prices, say the writers of a recent opinion piece published in the New England Journal of Medicine. CMS price negotiation under the Inflation Reduction Act will not have any immediate effect on gene therapy prices and may never have.

CSL Behring's Hemgenix may have some competition. At least three gene therapies for hemophilia B are in development.

STAQ Pharma has stepped in to supply desmopressin nasal spray for people with hemophilia A or Type 1 von Willebrand disease. The company is also producing albuterol for children’s hospitals.

STAQ, a 503B outsourcing facility, started making desmopressin nasal spray after Ferring Pharmaceuticals suspended production nearly three years ago.

Hemgenix for hemophilia B is priced to cost $3.5 million per treatment. Roctavian for hemophilia A may be approved by the FDA in July and is likely to be priced to cost between $2 million and $3 million per treatment.

Patient groups say the insurer is cutting off access to important medications. The Tennessee Blues plan say many of the drugs that it excluded from coverage have zero utilization.

The new factor VIII product is part of a surge of research and drug development for hemophilia.

Payers are bracing themselves for more ultra-expensive gene therapies.

German researchers developed a modified adeno-associated virus capsid that has greater affinity for liver cells.

Enyzyre says its at-home testing system will be a major advance for people with hemophilia because they will better able to manage the disease on their own.

CSL Behring's gene therapy, called EntranaDez, may be approved later this month. BioMarin, Pfizer, Bayer and Freeline Therapeutics also have gene therapies for hemophilia in development.

The hemophilia organization developed a shared decision-making tool for patients and providers.

Long-acting recombinant replacement factor gets favorable rating from multidisciplinary group. Gene therapy was viewed with some wariness.

Findings suggest that screening people with hemophilia for diabetes and hypertension might have benefits.

An analysis found that a single dose of gene therapy would be more cost-effective than on-demand or prophylaxis factor replacement in 92% of cases.

Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.

Lack of blood clotting may afford some protection against common cardiovascular disease events, such as ischemic stroke.

A small study finds that an adeno-associated virus gene therapy eliminated both spontaneous bleeding and the need for factor IX prophylaxis in patients with hemophilia B.

ICER said Bluebird Bio could use an outcome-based pricing plan of five yearly payments totaling $2.1 million for patients treated with beti-cel who no longer need blood transfusions.

Study adds to the evidence that reducing the risk of blood clots should be one of the main goals of managing these myeloproliferative disorders. Monitoring cholesterol levels may be especially important.

Longer half-lives mean less frequent administration and improved quality of life.