Pharma Focus Europe

Prilenia Plans to Seek Marketing Authorization Application (MAA) Approval for Pridopidine in Huntington’s Disease from the EU

Wednesday, March 13, 2024

Prilenia Therapeutics B.V., a company specializing in the development of innovative therapies for neurodegenerative diseases and neurodevelopmental disorders, has announced its intention to submit a Marketing Authorization Application (MAA) for pridopidine to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for the treatment of Huntington’s disease (HD). This decision comes after successful pre-submission discussions with regulatory authorities in the European Union, with the submission planned for mid-2024.

Dr. Michael R. Hayden, CEO of Prilenia, emphasized the consistent therapeutic benefits of pridopidine across various important measures for HD patients and their families. These measures include daily functioning, cognition, motor skills, and overall clinical progression, particularly in HD patients not using anti-dopaminergic medications (ADMs).

"We are appreciative of the constructive discussions with European regulators regarding our pridopidine data," said Dr. Hayden. "This paves the way for potential approval of a therapy for HD, a rare neurodegenerative disease with a predictable decline and a lack of approved treatments targeting disease progression. We are now finalizing our MAA submission for mid-2024 and preparing for pridopidine's potential commercial availability for patients in Europe."

Prilenia also intends to engage with the U.S. Food and Drug Administration (FDA) to discuss pridopidine's potential as a treatment for HD in the United States. Additionally, the company plans to consider submitting regulatory applications for other countries and regions globally following the European regulatory review process.

In January 2024, Prilenia announced completion of discussions with global regulatory agencies for pridopidine's development in amyotrophic lateral sclerosis (ALS), planning a single pivotal Phase 3 study.

Pridopidine is an oral S1R agonist administered at 45 mg twice daily, exhibiting a safety and tolerability profile comparable to placebo in clinical studies involving approximately 1,700 patients over seven years. The S1R protein, highly expressed in the brain and spinal cord, regulates key processes commonly impaired in neurodegenerative diseases. Activation of S1R by pridopidine stimulates protective cellular pathways, including enhancing autophagy, axonal transport, mitochondrial energy production, and restoring calcium homeostasis, all crucial for neuronal function and survival, leading to neuroprotective effects.

Prilenia has received Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU, along with Fast Track designation from the U.S. FDA for HD treatment.

Huntington’s disease is a rare inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. It affects approximately 100,000 people worldwide, with an additional 300,000 individuals at risk. HD leads to the degeneration of neurons in the brain, resulting in functional, motor, cognitive, and behavioral symptoms, with no current treatments addressing disease progression.

PROOF-HD, a Phase 3 study evaluating pridopidine's safety and efficacy in HD patients, demonstrated sustained benefits across multiple clinically relevant endpoints, with a safety profile comparable to placebo.

In conclusion, Prilenia's progress with pridopidine represents a significant step forward in addressing the urgent need for effective therapies for HD and other neurodegenerative disorders.



Thermo Fisher Scientific - mRNA ServicesWorld Orphan Drug Congress 2024World Vaccine Congress Europe 2024Advanced Therapies USA 2024