FDA grants breakthrough therapy designation to Praxis’ relutrigine for pediatric epilepsy

Published 17/07/2025, 13:26
FDA grants breakthrough therapy designation to Praxis’ relutrigine for pediatric epilepsy

Praxis Precision Medicines, Inc. (NASDAQ:PRAX), currently valued at $1.1 billion, announced Thursday that the U.S. Food and Drug Administration has granted breakthrough therapy designation to its investigational drug relutrigine for pediatric use in treating patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The company said the designation was supported by positive data from cohort 1 of its Phase 2 EMBOLD study and from 11 months of open-label extension data. According to InvestingPro data, the stock has shown strong momentum, delivering impressive returns over the past three months despite its characteristic volatility.

The breakthrough therapy designation is intended to expedite development and regulatory review for drugs that may demonstrate substantial improvement over existing therapies for serious conditions, according to the FDA. Relutrigine is described as a sodium channel functional state modulator.

Praxis also stated that it has initiated the EMERALD study, which will evaluate relutrigine in patients across all developmental and epileptic encephalopathies.

In addition, Praxis reiterated that it has commenced an investor quiet period ahead of the anticipated topline results from the RADIANT Phase 2 study, which are expected by mid-year 2025. The quiet period is expected to last up to three weeks.

This information is based on a press release statement included in the company’s Form 8-K filing with the Securities and Exchange Commission.

In other recent news, Praxis Precision Medicines has been granted Breakthrough Therapy Designation by the FDA for its drug relutrigine, intended to treat SCN2A and SCN8A developmental and epileptic encephalopathies. This designation follows promising results from the Phase 2 EMBOLD trial, which showed significant seizure reduction in patients. Piper Sandler has maintained its Overweight rating on Praxis, with a price target of $270, emphasizing the company’s promising epilepsy treatments, including vormatrigine, which is expected to provide key data by mid-2025. Oppenheimer also assigned an Outperform rating to Praxis, citing optimism about the company’s financial stability and strategic direction, with cash reserves reported at $472 million. The company is advancing its relutrigine and vormatrigine programs, with several trials underway, including the EMERALD and RADIANT trials. Meanwhile, the Prax Group, unrelated to Praxis Precision Medicines, has entered insolvency proceedings, impacting its operations in the UK energy sector, including the Lindsey oil refinery. This development highlights the challenges faced by the Prax Group amidst mounting losses. These recent developments underscore significant movements in both the pharmaceutical and energy sectors.

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