PTC Therapeutics Announces Commercial Milestone Payment Following the Recent European Approval of Evrysdi™
SOUTH PLAINFIELD, N.J., April 1, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that a $20 million milestone payment was triggered by the first commercial sale of Evrysdi™ (risdiplam) in the European Union under its License and Collaboration Agreement with Roche. Approval for Evrysdi from the European Medicines Agency was received on March 30 for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older.
"We are happy to see the rapid adoption of Evrysdi in the EU which speaks to the need for new treatments for SMA patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "We are delighted that an effective at-home therapy will be available to SMA patients. We recognize that a large proportion of SMA patients in the EU are currently not receiving an approved therapy."
Roche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access to SMA patients. Evrysdi is immediately accessible to patients in Germany and will be accessible from early April to patients in France through the cohort Temporary Authorization for Use. Evrysdi has currently been approved in 38 countries and submitted for Health Authority review in a further 33 countries.
Evrysdi is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche led the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
About Spinal Muscular Atrophy (SMA)
About Evrysdi™ (risdiplam)
About the Evrysdi™ (risdiplam) Clinical Studies
FIREFISH (NCT02913482) is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years followed by an open-label extension. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) (defined as sitting without support for five seconds). The study met its primary endpoint.
SUNFISH (NCT02908685) is a two part, double-blind, placebo controlled pivotal study in people aged 2 to 25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the Motor Function Measure 32 (MFM-32) scale at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
Clinical Trial Safety Data
In addition to FIREFISH and SUNFISH, Evrysdi™ is being evaluated in a broad range of people with SMA, including in:
JEWELFISH (NCT03032172) is an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi™. The study has completed recruitment (n=174).
RAINBOWFISH (NCT03779334) is an open-label, single-arm, multi-center study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to 6 weeks old (at first dose), with genetically diagnosed SMA, who are not yet presenting symptoms. The study is currently recruiting.
About PTC Therapeutics, Inc.
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PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; the enrollment, conduct, and results of ongoing studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any regulatory submissions and commercialization with respect to Evrysdi; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Evrysdi.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
SOURCE PTC Therapeutics, Inc.
Company Codes: NASDAQ-NMS:PTCT