The FDA created its Orphan Drug Act to incentivize drug developers to develop solutions for patients with rare or life-threatening conditions. Now, Orphan Drug designation is granted to drugs or biologics designed to treat these rare conditions, defined as affecting under 200,000 Americans. According to a recent press release from Catalyst Biosciences, Inc. (“Catalyst”), the company’s drug candidate MarzAA (subcutaneous Marzeptacog alfa [activated]) recently earned Orphan Drug designation for the treatment of patients with Factor VII deficiency.
I first wrote about MarzAA in May 2021, when Catalyst announced that the first patient was dosed in a Phase 1/2 clinical trial evaluating the treatment for patients with a variety of bleeding disorders, including Glanzmann thrombasthenia and Factor VII deficiency. Since late 2020, the treatment has earned a variety of accolades: Orphan Drug and Fast Track designations for hemophilia A/B, as well as Fast Track designation for Factor VII deficiency.
Now, MarzAA has earned another Orphan Drug designation for the treatment of patients with Factor VII deficiency. MarzAA is an engineered coagulation Factor VIIa (FVIIa) which is administered to patients subcutaneously. So far, the treatment has shown promise and efficacy in patients with bleeding disorders across a variety of clinical and preclinical studies.
Because Catalyst’s product earned Orphan Drug designation, the company also receives benefits. With Orphan Drug designation comes fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval.
Factor VII Deficiency (FVIID)
Factor VII deficiency (FVIID) is a rare bleeding disorder characterized by deficient or low-activity clotting factor VII. F7 gene mutations cause this condition. Normally, F7 provides instructions to make a protein called coagulation factor VII. However, the gene mutations prevent the body from creating enough factor VII – or from having the protein work the correct way. As a result, the blood does not clot normally. Patients with FVIID may experience excessive bleeding. Symptoms are typically more severe the earlier someone is diagnosed. When symptoms appear, these include:
- Easy bruising and bleeding
- Prolonged bleeding during menstruation, after injury, or after surgery
- Frequent nose bleeds
- Bleeding of the gums
- Hematuria (blood in the urine)
- Black, tarry stool
- Joint pain if bleeding into joint spaces
- Reduced range of mobility
- Intracranial hemorrhage