EU agency say no trials of mesdopetam in children are needed
Irlab granted waiver, will advance other studies of Parkinson's therapy
The European Medicines Agency (EMA), which evaluates the safety and efficacy of new drugs in the European Union, has waived a requirement that would have had Irlab Therapeutics conducting clinical trials testing mesdopetam — its therapy candidate for levodopa-induced dyskinesia, or uncontrolled, involuntary movements common in Parkinson’s disease patients — in children.
The decision follows the recommendation of the EMA’s Pediatric Committee and frees the company from the obligation of submitting pediatric investigation plans for the new drug. Such testing would otherwise be compulsory before an EU marketing authorization application could be submitted.
The waiver recommendation may be given to pharmaceutical companies in cases in which developing a certain drug for children is not appropriate or feasible.
“We are pleased that EMA has confirmed that pediatric studies evaluating mesdopetam are not needed to support a market authorization application for Parkinson’s disease,” Kristina Torfgård, PhD, Irlab’s CEO, said in a company press release. According to Torfgård, Irlab already had received a similar decision from the U.S. Food and Drug Administration (FDA).
Developer planning Phase 3 trial of mesdopetam in adults
Parkinson’s is caused by the progressive loss of dopaminergic neurons, which are the nerve cells responsible for producing the signaling molecule dopamine. Reduced dopamine signaling ultimately gives rise to most of the disease’s symptoms.
The mainstay treatment for Parkinson’s is levodopa, a medication that provides a molecule that cells can use to produce dopamine. Although levodopa is effective at easing Parkinson’s motor symptoms, its long-term use frequently leads to dyskinesia, or uncontrolled movements, and so-called off periods, or times when symptoms are not well controlled between doses.
Mesdopetam, formerly IRL790, is an Irlab therapy that’s designed to block the activity of a dopamine receptor called D3, which has been linked with levodopa-induced dyskinesia.
In a Phase 2b clinical trial (NCT04435431) in adults, the treatment’s higher dose — 7.5 mg, given twice daily — was shown to ease levodopa-induced dyskinesia relative to a placebo. It increased patients’ good on time and reduced off times and dyskinesia severity compared with the placebo. Good on time is defined as periods in which symptoms are well-controlled without dyskinesia, while off times are periods when symptoms are not well-controlled.
Since we have previously received a corresponding decision from the … [FDA], the decision from EMA means we can now focus our development efforts entirely on activities in more relevant patient groups.
The company is now preparing a Phase 3 clinical trial to test mesdopetam’s efficacy in adults with Parkinson’s who have at least two daily hours of dyskinesia. Participants will receive the treatment at a dose of 7.5 mg, twice daily, or a placebo, for about three months.
Besides its anti-dyskinetic effects, mesdopetam also has been shown to reduce signs of psychosis in a mouse model of Parkinson’s. Psychosis is a common nonmotor symptom of the disease, characterized by hallucinations — seeing, hearing, or feeling things that do not exist — and/or delusions.
According to Torfgård, the EMA decision will allow Irlab to better advance the experimental therapy.
“Since we have previously received a corresponding decision from the … [FDA], the decision from EMA means we can now focus our development efforts entirely on activities in more relevant patient groups,” Torfgård said.
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