FDA Grants Rare Pediatric Disease Designation to Cerecor’s Treatment for Lymphatic Malformations

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Cerecor’s CERC-006 for the treatment of lymphatic malformations.

Lymphatic malformations (LMs) are rare, non-malignant masses consisting of fluid-filled channels or spaces thought to be caused by the abnormal development of the lymphatic system. LM occurs mostly in infancy or early childhood and can persist throughout life. It is estimated that 30,000 to 60,000 people have this condition in the United States.

“This designation underscores the high unmet need in this family of serious and rare diseases of infants and children that so often lead to disability and in some cases, death,” said Mike Cola, CEO of Cerecor.

CERC-006 is an orally available blocker of mTOR complex 1 and 2 that is being developed for the treatment of serious lymphatic malformations not treatable with surgery or sclerotherapy. Because a large majority of LM patients have activating mutations in the PK/AKT/mTOR pathway, the company believes CERC-006 has the potential to specifically reduce proliferation of the abnormal cells that cause LM, reduce the size of neoplastic lesions and restore lymphatic function; ultimately improving and prolonging the lives of many affected children. A phase 1b/2a proof-of-concept trial to test the safety and efficacy of CERC-006 in LM is planned to begin in 2021.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes CERC-006 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

Photo: Mike Cola, CEO of Cerecor