FRANKFURT, Germany – Bayer announced Monday that the European Commission has approved Kovaltry, a drug for the treatments of patients who suffer from haemophilia A, Reuters reported.
The German multinational chemical and pharmaceutical company said the drug will complement its existing haemophilia business, but Kovaltry marks the most important product.
Haemophilia is a recessive disorder that affects an estimated of 400,000 people globally. It is caused by deficiency of proteins needed to form blood clots and could be inherited or arise from spontaneous mutation, according to Medscape.
Bayer’s new drug is an unmodified full-length recombinant factor VIII product. It has been successfully tested in haemophilia A patients, controlling and protecting them from bleeds when applied prophylactically up to three times per week.
Regulators approved Kovaltry following the positive findings from the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Haemophilia A Disease) Clinical Development Program. Three clinical trials were carried out, showing Kovaltry’s pharmacokinetics, safety and efficacy.
For example, researchers found with LEOPOLD II that patients taking the drug twice a week in the prophylaxis arm presented a 93.3% reduction in median annualized bleeding rate. Findings also showed a 96.7% reduction in the three-times-per-week arm.
The medicine is designed for patients from all age groups.
Symptoms of haemophilia
Patients may present diverse symptoms depending on their specific levels of FVIII activity. The signs of mild haemophilia include easy bruising, traumatic injury and inadequate clotting. In the most severe cases patients may present spontaneous hemorrhage.
The usual symptoms are weakness, tachycardia, orthostasis and tachypnea. Patients may also present musculoskeletal signs such as cracking, pain, stiffness and tingling, as well as gastrointestinal symptoms like abdominal pain, stiff neck and frank red blood per rectum.