Ultragenyx says EMA validates Sanfilippo gene therapy application
Rebisufligene etisparvovec won U.S. approval on Sept. 17; Ultragenyx gave no date for a European decision.
Adrian Kessler
The EMA can begin scientific review of a gene therapy for Sanfilippo syndrome type A after Ultragenyx Pharmaceutical Inc. NASDAQRARE said on Oct. 2 that the agency validated its application for rebisufligene etisparvovec. The European filing follows the drug's Sept. 17 U.S. approval, but the release gave no date for a decision in Europe.
Validation means the dossier is complete enough for scientific review to start, according to the company-attributed release. It makes no finding about the therapy's safety or efficacy and does not grant European marketing authorisation.
Ultragenyx is a California rare-disease drugmaker with marketed medicines and a clinical gene-therapy pipeline. Its U.S. approval of this treatment gives investors a commercial milestone to follow as it pursues European authorisation for the same therapy.
On Oct. 2, Ultragenyx's NASDAQRARE shares rose 3.19 per cent to US$15.22 at the 4 p.m. EDT Nasdaq close. The company did not attribute that day's trading to the European application in its release.
EMA Validation Allows Scientific Review to Begin
Ultragenyx said it submitted the marketing authorisation application to the EMA for treatment of mucopolysaccharidosis type IIIA, the inherited disease also called Sanfilippo syndrome type A. The application asks for permission to market the therapy in Europe; validation confirms the dossier is sufficiently complete for scientific assessment to begin.
The treatment is a single-dose intravenous AAV9 gene therapy designed to deliver a working copy of the gene for sulfamidase, an enzyme missing in patients with the disease. Without that enzyme, heparan sulfate accumulates in cells and the disease damages the central nervous system, the company said in its Oct. 2 release.
“The validation of our application by the EMA marks an important regulatory milestone in the ongoing review process for this investigational therapy,” Ultragenyx senior vice-president Jane Cooper said in the Oct. 2 release.
The same release says the therapy has EMA PRIME and orphan-drug designations. Those designations support development and assessment, but neither represents a marketing decision. Ultragenyx also said the application will fall within the scope of the EU's Joint Clinical Assessment framework, which is intended to coordinate clinical assessment across EU member states.
The EMA publishes monthly lists of medicines under evaluation; its latest list available when this story was checked was dated Sept. 9, before the company's Oct. 2 announcement. Ultragenyx did not specify a date for a committee opinion or a subsequent European authorisation decision.
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U.S. Product Is Expected to Be Available for Shipment
The Sept. 17 U.S. approval announcement identified the approved pediatric therapy as FAYUVI. That announcement said the pivotal analysis compared 17 treated patients with 27 untreated patients from an external natural-history cohort. The treated group had a mean cognitive score 23.5 points higher over the study period, with a p value below 0.0001, according to the company.
Ultragenyx characterized the treatment's safety profile as acceptable in that release, which said follow-up extends to nearly eight years for some patients. The European regulator will make its own assessment of the submitted evidence, and the U.S. pediatric indication does not establish the eventual scope of any European authorisation.
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For sector context, the iShares Biotechnology ETF NASDAQIBB closed at US$205.29 at 4 p.m. EDT on Oct. 2. Listed comparison REGENXBIO Inc. NASDAQRGNX closed at US$7.43 on Nasdaq at 4 p.m. EDT on Oct. 2.
Ultragenyx reported US$436 million in cash, equivalents and marketable securities at June 30 in its Aug. 4 quarterly release. It used US$97 million in operations during that quarter, before the Sept. 17 FAYUVI approval.
Ultragenyx expects commercial FAYUVI product to be available for shipment to qualified U.S. treatment centres within 30 to 60 days of its Sept. 17 approval, according to its approval announcement. That window will test whether the company has made product ready to ship; no date has been announced for a European decision.
