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Rare Pediatric Disease designation granted to ‘lonafarnib’ for Hutchinson-Gilford Progeria Syndrome

Hutchinson-Gilford Progeria Syndrome, progeroid laminopathies

The drug 'lonafarnib' has been granted Rare Pediatric Disease (RPD) designation by The U.S. Food and Drug Administration (USFDA) for the treatment of both Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies.

David Cory, president and chief executive of Eiger expressed satisfaction by the Rare Pediatric Disease designation grant to 'lonafarnib' for the ultra-rare and fatal genetic pediatric condition in which accelerated ageing is the cardinal feature. He sounded hopeful and encouraged by the agency’s support for lonafarnib and the opportunities provided by the Priority Review Voucher Program and Rare Pediatric Disease designation.

He revealed the company’s plan to submit an NDA for lonafarnib for the treatment of progeria and progeroid laminopathies in 2019. In the meantime, he shared, company has planned to ensure access to lonafarnib for all the eligible patients with these disorders, and launch an expanded access program by the end of year on a global scale.

Complete article can be read here