EMA adds Alnylam RNAi drug to PRIME fast-track scheme

EMA's offices

The European Medicines Agency (EMA) has added Alnylam’s acute hepatic porphyria candidate to its priority medicine (PRIME) scheme. Securing the status positions Alnylam to benefit from a close relationship with the EMA as it works to move the drug prospect into phase 3 by the end of the year.

Alnylam gained PRIME status for its aminolevulinic acid synthase 1 (ALAS1)-targeting RNAi therapy on the strength of data from an ongoing phase 1 study, in which it has shown the potential to cut the number of porphyria attacks in patients with acute intermittent porphyria (AIP). The genetic condition is associated with attacks that cause hospitalization. And, while Recordati Rare Diseases’ Panhematin is used to manage symptoms, there is a lack of preventative options.

The combination of promising early data and a lack of approved therapeutic alternatives puts the Alnylam drug, givosiran, in the sweet spot of the EMA scheme. The EMA set up the scheme to support development of drugs that target unmet medical needs by providing a dedicated contact, scientific advice and the potential for accelerated assessment to drug developers.

PRIME is more selective than some other regulatory schemes, such as the orphan drug program that givosiran was previously accepted into. At the last count, EMA had accepted 17 drugs into PRIME, while rejecting 60 and deeming a further five to be out of scope. One-third of the successful applications covered oncology drugs.

By becoming a rare example of an ex-oncology PRIME program, Alnylam has positioned itself to receive extra attention from the EMA at a critical point in the development of givosiran.

“We look forward to collaborating with the EMA on the accelerated assessment of givosiran, with the goal of advancing this investigational medicine into a phase 3 trial in late 2017,” Alnylam VP Jeff Miller said in a statement.

Givosiran is designed to improve outcomes in AIP and other forms of acute hepatic porphyria by inhibiting the liver-expressed enzyme ALAS1, which, in turn, slows the accumulation of the heme intermediates that cause symptoms.