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news.Eiger BioPharmaceuticals has secured the Rare Pediatric Disease (RPD) designation from the US Food and Drug Administration (FDA) for lonafarnib to treat both Hutchinson-Gilford Progeria Syndrome (HGPS or progeria) and progeroid laminopathies.
Image: The US FDA’s Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration.
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RPD designation for progeria and progeroid laminopathies enables priority review voucher (PRV) eligibility upon FDA approval of a rare pediatric disease product application for lonafarnib for these ultra-rare and fatal genetic conditions characterized by accelerated aging in children.
Eiger is collaborating with The Progeria Research Foundation in this lonafarnib program and plans to submit a new drug application (NDA) to the FDA in 2019. There is no approved treatment for progeria or progeroid laminopathies.
The Priority Review Voucher Program is focused on encouraging development of therapies to prevent and treat rare pediatric diseases. If an NDA for lonafarnib is approved by the FDA for progeria and progeroid laminopathies, the RPD designation qualifies Eiger, as the NDA sponsor, for a PRV upon marketing approval.
The voucher, which can be sold or transferred to another entity, can be used by the holder to receive priority review for a future NDA or BLA submission, which reduces FDA’s target submission review time from ten months to six months. Eiger would share 50% of the proceeds from monetization of a Priority Review Voucher with PRF to support future progeria research.
Eiger president and CEO David Cory said: “We are encouraged by the agency’s support for lonafarnib and the opportunities provided by the Rare Pediatric Disease designation and the Priority Review Voucher Program.
“We plan to submit an NDA for lonafarnib in the treatment of progeria and progeroid laminopathies in 2019. In the meantime, we are committed to ensuring access to lonafarnib for all patients with these disorders, and plan to launch a global expanded access program by end of year.”
Source: Company Press Release
