Regulatory Updates

A roundup of recent news around designations, approvals and patent activities globally

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Zafgen granted Orphan Drug designation in EU for beloranib
CAMBRIDGE, Mass.—Zafgen Inc., a biopharmaceutical company dedicated to improving the health and well-being of patients affected by obesity, announced in July that the European Commission (EC) had granted orphan drug designation for beloranib for the treatment of the rare disease Prader-Willi syndrome (PWS) in the European Union.
“We are very pleased that our application for orphan drug designation of beloranib has been positively reviewed and granted by the EC,” said Dr. Thomas Hughes, CEO of Zafgen. “On the basis of our Phase 2 results reported earlier this year, we believe beloranib represents a promising new approach for the treatment of PWS, with the potential to meaningfully improve the lives of patients with this severe and life-threatening condition. We are dedicated to the advancement of beloranib for the treatment of PWS and other severe forms of obesity, and we look forward to initiating our Phase 3 clinical program in PWS later this year.”
Initial results from the company’s Phase 2 study of beloranib in patients with PWS reportedly demonstrated improvements in hunger-related behaviors and body composition, including reductions in body fat content and preserved lean body mass.
In January 2013, the U.S. Food and Drug Administration granted Zafgen Orphan Drug designation to treat PWS with beloranib.

Pharmalink’s core patents for Nefecon granted
STOCKHOLM, Sweden—Pharmalink AB, a specialty pharma company focused on orphan and niche products, reported July 10 that core patents for its late-stage clinical candidate Nefecon were issued in the key markets of the United States, Europe, China and Hong Kong. A patent is pending in Japan.
Nefecon is a potential disease-modifying treatment for patients with primary IgA nephropathy at risk of developing end-stage renal disease. Nefecon has shown positive results in an open-labelled Phase 2 trial evaluating safety and efficacy and is now being tested in a placebo-controlled randomized Phase 2b study. Pharmalink recently completed patient enrolment of this study, which has been designed to enable an optimal dose of Nefecon to be selected for a Phase 3 registration trial. Headline data is anticipated in the third quarter of 2015.
The patents issued provide protection around the formulation of Nefecon and its use as a treatment of glomerulonephritis, including IgA nephropathy, the most common form of primary glomerulonephritis and a cause of end-stage renal disease. The formulation technology, TARGIT, was licenced by Pharmalink for exclusive use with Nefecon under a 2011 agreement with Archimedes Development Ltd. in the United Kingdom.
“Nefecon is a disease-modifying medication and one of the first specifically aimed at addressing the underlying pathology of progressive IgA nephropathy,” said Johan Häggblad, managing director of Pharmalink. “The issuance of core patents in key global markets reinforces the commercial package we are building around Nefecon.”

PTC Therapeutics receives conditional approval in EU for Translarna
SOUTH PLAINFIELD, N.J.—On Aug. 4, PTC Therapeutics Inc. announced that the European Commission had granted conditional marketing authorization for Translarna (ataluren) in the European Union (EU) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
“We are delighted that Translarna was approved for the treatment of nonsense mutation Duchenne muscular dystrophy. By targeting the underlying cause of DMD, it has the potential to change the course of the disease. We are moving rapidly to make this product available to patients in the EU as we continue our global efforts to fulfill our vision of making Translarna available to all the boys it may benefit,” stated Dr. Stuart W. Peltz, CEO of PTC Therapeutics. “We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. The DMD community has been waiting a long time for treatment options, and this conditional approval marks an important day for us all.”
The authorization allows PTC to market Translarna in the 28 countries that are member states of the EU, as well as European Economic Area members Iceland, Liechtenstein and Norway. As part of the conditional marketing authorization, PTC is obligated to complete its confirmatory Phase 3 trial in nmDMD (ACT DMD) and submit additional efficacy and safety data from the trial.

IBA receives FDA approval for Proton Therapy Specific Cone Beam CT
LOUVAIN-LA-NEUVE, Belgium—IBA (Ion Beam Applications SA), the world’s leading provider of proton therapy solutions for the treatment of cancer, announced in July that it had received combined clearances from the U.S. Food and Drug Administration (FDA) that will enable IBA to market-launch the Proton Therapy specific Cone Beam Computed Tomography (CBCT) solution.
As a component of IBA’s Image Guided Proton Therapy (IGPT) solution, CBCT provides 3D imaging for increased accuracy in patient treatment. IBA’s first CBCT is at the validation phase and the first clinical use is expected for the second half of 2014.
“We are delighted to be the first company to receive Marketing Authorization from the FDA for the Proton Therapy specific CBCT technology, said Olivier Legrain, CEO of IBA. “The development of imaging capabilities is critical to the expansion of the use of proton therapy in new cancer indications and IBA has developed advanced radiation therapy solutions that improve the overall treatment experience for both clinicians and patients. CBCT is just one example of the latest technological advancements achieved by IBA as we maintain our unrivalled position as the world’s leader in the innovation and delivery of proton therapy.”

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