PIM designation is an early indication that cenobamate is a promising candidate for the MHRA’s Early Access to Medicine Scheme
Milestone further supports the potential of cenobamate as an innovative approach to treat drug-resistant focal-onset seizures in adults in Europe
Zug, Switzerland, 11 August 2020 – Arvelle Therapeutics, an emerging biopharmaceutical company focused on bringing innovative treatments to patients suffering from CNS disorders, today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has informed the company that a Promising Innovative Medicine (PIM) designation has been issued for its investigational anti-seizure medication, cenobamate, for the treatment of drug-resistant focal-onset seizures in adults.
A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS). Launched in 2014, EAMS aims to provide patients in the UK with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
Commenting on the announcement, Stuart Mulheron, General Manager UK & Ireland, Arvelle Therapeutics said: “The MHRA PIM designation marks an important milestone for Arvelle. Together with the European Medicines Agency’s recent acceptance of the Marketing Authorization Application and the FDA’s approval of cenobamate in the US, it further supports the potential of cenobamate as an innovative treatment approach for the estimated 1.6 million people with epilepsy in Europe who are still experiencing seizures despite taking anti-seizure medications.”
The global disease burden of epilepsy is high, as a diagnosis of epilepsy confers significant disability on the individual, including physical, psychological and social problems that negatively impact self-esteem, family, relationships, leisure, work, career prospects and ability to drive.1,
People with epilepsy whose seizures are poorly controlled have higher morbidity and mortality rates and often experience comorbid illnesses, social stigmatisation, psychological dysfunction and reduced quality of life.,
Whilst reduction in seizure frequency brings benefits to patients, seizure freedom is the treatment outcome that has the unique potential to significantly impact the disease burden associated with epilepsy.4,,
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About Arvelle Therapeutics International GmbH
Arvelle Therapeutics International GmbH (“Arvelle”) is an emerging biopharmaceutical company focused on bringing innovative treatments to patients suffering from CNS disorders. Arvelle is headquartered in Zug, Switzerland.
In February 2019 Arvelle received start-up financing of $207.5 million, one of the largest initial financing commitments for a European-focused biopharmaceutical company from a global syndicate including NovaQuest Capital Management, BRV Capital Management, LSP, H.I.G. BioHealth Partners, Andera Partners and F-Prime Capital and KB Investment.
On receipt of this financing, Arvelle acquired exclusive European rights from SK Biopharmaceuticals for the development and commercialization of cenobamate, an investigational anti-seizure medication (ASM). To date, cenobamate has been approved by the US Food and Drug Administration (FDA) for the treatment of partial-onset (focal) seizures in adults, and is commercially available in the U.S.
In March 2020, Arvelle announced the European Medicines Agency’s (EMA) acceptance of the marketing authorization application (MAA) for cenobamate for the adjunctive treatment of focal-onset seizures in adults with epilepsy. This validation of the MAA confirms Arvelle’s application is complete and marks the start of the assessment process for cenobamate.
Cenobamate was discovered by SK Biopharmaceuticals and SK life science and is a new FDA-approved anti-seizure medication (ASM) for the treatment of partial-onset seizures in adults (also known as focal-onset seizures). Cenobamate has been approved in the U.S. where it is commercially available under the trademark XCOPRI®. In early 2019, SK Biopharmaceuticals entered into an exclusive licensing agreement with Arvelle Therapeutics to develop and commercialize cenobamate in Europe.
Cenobamate is believed to deliver its dual, complementary mechanism of action through a combination of two mechanisms: enhancing inhibitory currents through positive modulation of GABA-A receptors at a non-benzodiazepine binding site, and decreasing excitatory currents by both inhibiting the persistent sodium current and enhancing the inactivated state of voltage-gated sodium channels.
As a once-a-day tablet, cenobamate has been studied in over 2,500 subjects, including more than 1,900 patients across the two phase 2 and one phase 3 trial. Seizure freedom rates seen in the double-blind placebo controlled pivotal studies, of adult patients with drug-resistant focal onset seizures who were administered cenobamate as an adjunctive therapy have exceeded existing supportive care.,
Long-term data of cenobamate is being studied in the open-label extensions of the double-blind placebo-control trials as well as the open-label safety study in adults with uncontrolled focal-onset seizures. Additionally, cenobamate is being assessed in an ongoing randomized, double-blind, placebo-controlled trial evaluating its safety and efficacy as adjunctive therapy in patients with primary generalized tonic-clonic seizures (NCT03678753).
About the MHRA Early Access to Medicine Scheme
The MHRA Early Access to Medicines Scheme aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. The MHRA will give a scientific opinion on the benefit/risk balance of the medicine, based on the data available; this opinion does not replace the normal licensing procedures for medicines but supports the prescriber and patient to make a decision on whether to use the medicine before its licence is approved.
There are two parts to the scheme. Step 1 is to designate a product with a “promising innovative medicine” (PIM) designation. This gives an indication that a product may be eligible for the Early Access to Medicines Scheme (based on early clinical data). In Step 2 the MHRA issues a scientific benefit/risk opinion, following the assessment of quality, safety and efficacy data. This describes the benefits and risks of the medicine, based on the information submitted to MHRA by an applicant after sufficient data have been gathered from clinical trials and other studies.
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. Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Cenobamate Adjunctive Therapy in PGTC Seizures NCT03678753.