FDA shoots down Lykos' MDMA therapy for PTSD
The FDA has denied Lykos Therapeutics' marketing application for its post-traumatic stress disorder (PTSD) therapy based on MDMA, also known as ecstasy, in a setback for the emerging psychedelic medicines category.
The rejection had been well signposted, given that an FDA advisory committee voted nine to two in June that the data behind Lykos' midomafetamine candidate was not strong enough to support approval and the influential Institute for Clinical and Economic Review (ICER) had poked holes in the design of the main clinical trial the company was relying on in its application.
The FDA has requested Lykos conduct another phase 3 trial, although, the company said it planned to ask for a meeting to ask the agency to reconsider that stance, whilst also seeking a way forward for the programme.
As the first Schedule 1 psychedelic medicine to go through the FDA review process – in other words, based on a drug with a high potential for abuse and no defined medical effect – there has been a lot of attention to midomafetamine, which is intended for use in tandem with psychotherapy.
Still, market watchers have suggested there should be no read-through to other psychedelic medicines coming through late-stage development, as the decision is very much centred on deficiencies in Lykos' application.
Lykos said in a statement that, in its complete response letter (CRL), the FDA had followed the lead of its advisors in concluding that the data in support of a clinical effect was limited, and questioning whether "psychotherapy contributes to treatment benefit and if it is even necessary."
Chief executive Amy Emerson said that the FDA's request for a new trial is "deeply disappointing, not just for all those who dedicated their lives to this pioneering effort, but principally for the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades."
The company has taken issue with the make-up of the Psychopharmacologic Drugs Advisory Committee (PDAC), claiming that it had a limited number of "subject matter experts" and "veered beyond the scientific content" of the FDA's briefing documents.
The PDAC had numerous concerns about the data, including the potential for bias due to "functional unblinding" of the study, problems with data collection, uncertainties about the efficacy of the therapy in the longer term, and the risk of side effects, including cardiovascular and blood pressure problems and injury due to patient impairment, as well as abuse.
So far, Australia's Therapeutics Goods Administration (TGA) is the first global regulator to reschedule psychedelics (MDMA and psilocybin) for clinical use – specifically to assist psychotherapy sessions for PTSD, under carefully controlled conditions – although, some other countries including Canada and Switzerland allow compassionate use.
It is estimated that there are around 13 million people in the US suffering from PTSD, according to the FDA, which has granted breakthrough designation to midomafetamine in this setting.
Photo by Fatima Shahid on Unsplash
