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FDA Approves First Treatment for Rare Inherited Blood Disorder

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The U.S. Food and Drug Administration has recently approved Adzynma, the first recombinant (genetically engineered) protein product indicated for prophylactic or on demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) a rare and life-threatening blood clotting disorder.

Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

This condition is caused by a mutation in the ADAMTS13 gene, responsible for marking an enzyme with the same name that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body. Individuals with this condition can experience severe bleeding episodes, strokes and damage to vital organs, and can be fatal if left untreated.

Treatment of cTTP typically involves prophylactic plasma-based therapy for individuals with chronic disease to reduce the risk of clotting/bleeding by replenishing the absent ADAMTS13 enzyme. Adzynma is a purified recombinant form of the ADAMTS13 enzyme that works by providing a replacement for the low levels of the deficient enzyme in patients.

The safety and effectiveness of Adzynma were demonstrated in a global study evaluation prophylactic and on-demand enzyme replacement therapy with Adzynma compared to plasma-based therapies in patients with cTTP.

Orphan Designation and Rare Pediatric Priority Review Voucher

To support the development and evaluation of new treatments for rare disease, FDA grants orphan drug designation to drugs for the prevention, diagnosis, or treatment of conditions affecting fewer than 200.000 persons in the U.S. Offering benefits such as tax credits for qualified clinical trials, exemption from uses fees, and potential seven years of market exclusivity after approval.

Adzynma was designated an orphan medicine on July 2008. Also, the application was awarded a Rare Pediatric Disease Priority Review Voucher by the FDA for the development of rare pediatric disease products, meaning that the rare disease primarily affects individuals aged from birth to 18 years.  

Expedited Programs

This application was also granted Fast Track and Priority Review. Fast Track Designation can be applied for investigational new drugs that intends to treat a serious condition, and for which nonclinical or clinical data demonstrate the potential to address and unmet medical need in patients with such conditions.

Priority Review would include a product that was granted with Fast Track Designation and, if approved, would provide a significant improvement in the safety or effectiveness of the treatment of the condition. A Priority Review designation means FDA’s goal is to take action on the marketing authorisation application within 6 months (compared to 10 months under standard review).

If you are interested in FDA’s options for expedited programs, you can check our review on Expedited programs for regenerative medicine therapies for serious conditions here!  

Check the official FDA website if you want to know more or to consult the complete updated information related to the drug here.

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