Santhera Gets First Duchenne Drug OKd Under UK Early Access Scheme
Executive Summary
Santhera Pharmaceuticals’ Raxone has become the first treatment for Duchenne muscular dystrophy to be OKd under the UK’s early access to medicines scheme. The scheme enables patients to access the treatment ahead of it receiving an EU marketing authorization.
Santhera Pharmaceuticals has become the first company to have an unlicensed treatment for Duchenne muscular dystrophy (DMD) approved for use under the UK’s early access to medicines scheme (EAMS), meaning the therapy can be made available to UK patients while it undergoes review for marketing throughout the EU.
Raxone (idebenone) received a positive scientific opinion under the EAMS from the Medicines and Healthcare products Regulatory Agency, the regulator announced on June 21. The decision allows the drug to be used to slow the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids.
“We are able to provide Raxone for eligible patients on receipt of a prescription from a physician as soon as expert centres have received the necessary training for the EAMS,” Santhera told the Pink Sheet. “We are ready to provide that training now,” the company said.
While drugs under the EAMS are provided to the National Health Service free of charge, Santhera expects that the early access designation will help speed up the UK reimbursement review process for Raxone once it gains EU authorization for DMD, at which point the scientific opinion will expire. This will be of advantage to both the company and patients, it said.
As Raxone was granted a promising innovative medicine (PIM) status under the first stage of the two-stage EAMS process and now is made available under the EAMS via the subsequent scientific opinion stage, “we expect that NICE [the National Institute of Health and Care Excellence] will prioritise its review,” the company explained. “EAMS would also accelerate the implementation of a positive review by NICE once the drug is approved.”
EAMS products that subsequently receive marketing approval and are found to be cost effective following a health technology appraisal by NICE are commissioned by NHS England within 30 days of the NICE guidance, rather than the standard three months.
CHMP Opinion Expected In Q3 2017
An application to market the drug for DMD in the EU is currently being reviewed by the European Medicines Agency’s scientific committee, the CHMP. Raxone is already authorized in the EU for treating Leber’s hereditary optic neuropathy (LHON); the application for DMD was submitted as a Type II variation of the LHON marketing authorization.
“The assessors of the MHRA had full access to our correspondence with CHMP (and were very familiar with its content)” – Santhera
Santhera, which announced last month that it was expecting to receive a request from the CHMP for supplementary information to further support the clinical relevance of its data, predicts the committee might deliver an opinion on whether the European Commission should approve the application in Q3 2017.
“The exact same data [submitted to the CHMP] were submitted for the EAMS application,” the company told the Pink Sheet. “The assessors of the MHRA had full access to our correspondence with CHMP (and were very familiar with its content).”
The EU application is based on study data that demonstrated a statistically significant and clinically relevant benefit of Raxone in preserving respiratory function compared to placebo. This result is further substantiated by a natural history study, which shows that the benefits observed in the group treated with Raxone would not have been expected from the natural course of the disease, Santhera said.
No Other Treatment Available
The EAMS aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. DMD is a devastating genetic disorder characterized by progressive muscle degeneration that affects around one in every 3,500 boys born worldwide.
Patients with DMD who have progressed to the stage of respiratory decline, and who are not receiving glucocorticoids, have no treatment options available to them, other than supportive care, the MHRA said in a public assessment report for Raxone.
While PTC Therapeutics’ Translarna (ataluren) is authorized in the EU for treating DMD, the drug cannot be used in patients who are in respiratory decline, the agency noted. Ataluren is available for treating DMD patients with a so-called nonsense mutation in the dystrophin gene, but it is restricted to patients who are ambulatory, the MHRA said. DMD patients who are in respiratory decline will be largely unable to walk and will therefore not be eligible to receive ataluren.
“We’re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” Santhera Pharmaceuticals AG’s CEO, Thomas Meier, said in a statement. “This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”
As for Santhera’s experience with the EAMS, the company said that “in principle,” it would consider this option again for a late-stage, near-to-market product in a rare disease with high unmet medical need, though “at the present time” and in the “near future” it does not have any products in the pipeline that would qualify for an EAMS.
From the editors of Scrip Regulatory Affairs.