Will Elevidys Receive Approval from the European Medicines Agency (EMA)?

Patients with Duchenne muscular dystrophy (DMD) around Europe are anxiously awaiting the EMA's approval of Sarepta's application for its gene treatment Elevidys. Will Elevidys Receive Approval from the European Medicines Agency (EMA)?

Elevidys, a gene therapy developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD), is currently under review by regulatory bodies in different regions, including the European Medicines Agency (EMA). As of now, it is difficult to predict with certainty whether Elevidys will receive approval from the EMA, as the decision depends on various factors like clinical trial data, safety, efficacy, and the EMA’s assessment of the treatment’s overall benefit-risk profile.

In the U.S., the therapy has already been granted accelerated approval by the FDA, but for Europe, the review process is ongoing. The EMA will carefully assess the available clinical data before making its decision. While the FDA’s approval provides some optimism for Elevidys’ approval in other markets, each regulatory agency has its own process and standards for approval.

In Which Countries Is Elevidys Approved?

The only approved gene therapy for Duchenne muscular dystrophy in the world, Elevidys, was given accelerated approval in the US in June 2023 and is currently approved in the UAE, Qatar, Kuwait, Bahrain, Oman, Brazil, and Israel for the treatment of ambulant children with Duchenne who have a confirmed mutation in the DMD gene and are between the ages of 4 and plus.

In Which Countries Has Roche Applied for Authorization for Elevidys?

Applications for approval are currently under review in Europe (EMA), Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia.

Interim Results from Elevidys’ Embark Clinical Trial Could Impact Decision

Roche announced new EMBARK data showing significant and long-lasting benefits of Elevidys for outpatients with Duchenne muscular dystrophy (DMD). Positive news from ongoing studies could impact the EMA process, as many DMD patients see Elevidys as a source of hope. [Read More: New Results from the EMBARK Study]

Why Are Sarepta and Roche Only Applying for Registration in Certain Countries?

Sarepta is currently applying for approval for Elevidys in high-income countries such as Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia, raising concerns among DMD patients in financially disadvantaged countries. [Read More: Sarepta’s Revenue Expectations in 2025]

As DMD Warrior, we asked Sarepta and Roche which other countries they will apply for a license in, but we have not yet received a written response.

DMD patients expect the price of ELEVIDYS to be reduced as soon as possible and for registration applications to be made in all countries.

Families Concern if Sarepta for a Different Gene Therapy Will Not Be Approved by EMA

Many DMD patients worry that Duchenne gene therapies under development in Europe could negatively impact the EMA’s decision on Elevidys.

Many families are concerned that the greed for money in the health sector may impact the decisions to be taken.

The families want a lower price for Elevidys and a risk-sharing deal like the one in Brazil. [Learn More: Risk-Sharing Model]

Learn More: Potential Upcoming New Gene Therapies for DMD

Genethon and Sarepta Collaborated in 2017

The cost of Elevidys, which was developed by Sarepta and is known for its exorbitant price and the fact that it has not yet received approval from every country, has not yet been reduced.

In 2017, Genethon and Sarepta began a research partnership to complete the product’s preclinical development. With the co-development of the clinical gene therapy program utilizing this microdystrophin method, the agreement announced 2020 strengthens and broadens this partnership. [Read More: Genethon collaboration with Sarepta]

As per the deal, Sarepta will handle the rest of the world’s commercialization of the product GNT0004, while Genethon will handle it in Europe (except from the UK). Genethon has the right to royalties, development and commercial milestones, and an upfront payment from Sarepta. [Read: Genethon Gene Therapy GNT0004]

DMD patients are concerned that Genethon’s in-development GNT0004 gene therapy is as expensive as ELEVIDYS.

Learn More: FAQ about Elevidys

Pricing Policies Should Be Revised

A treatment method that is not easily accessible to patients will not be used by everyone, no matter how effective it is.

Everyone should be able to benefit from these treatments, not just those who are financially well-off.

For this reason, we are calling out to all DMD patients in the world who read this article. Reach out to Roche and Sarepta through all channels and request that they apply for a license in your country.

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