Laborie Medical Technologies Inc. continued a string of recent deals with an agreement to acquire Urotronic Inc. for $255 million cash up front with an additional $345 million in payments contingent on meeting specified commercial and reimbursement milestones. Laborie already held a minority interest in the manufacturer of the Optilume drug-coated balloon technology for treatment of urethral strictures and benign prostatic hyperplasia (BPH).
Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.
The FDA has awarded orphan drug designation to Healx Ltd.’s HLX-1502 for the treatment of neurofibromatosis type 1 (NF1). HLX-1502, discovered through the company’s proprietary artificial intelligence (AI) drug discovery platform, has a first-in-class mechanism and supporting data that indicate the potential for a good safety profile.
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
Now that the U.S. FDA has nearly 15 years of experience with developing and implementing a biosimilar pathway, it’s time for that regulatory path to catch up with the science, according to experts that have been involved in biosimilar development even before Congress passed the Biologics Price Competition and Innovation Act that created the framework for the U.S. biosimilar market.
The U.S. FDA’s concerns about the 510(k) program over the past decade-plus are practically the stuff of regulatory urban legend, but the agency has just unveiled a trio of draft guidances to address some of those concerns. Perhaps the most significant of these is a draft guidance for selection of a predicate device for a 510(k) filing, a document that may come across to industry as little more than an attempt to limit the devices that a manufacturer can recite in a 510(k) filing.
The U.S. FDA recently posted another round of recalls for the Cardiosave line of intra-aortic balloon pumps (IABPs) by Datascope Corp., advising users to make sure they have a backup unit at the ready in the case of device failure. However, the agency noted that “alternative treatments are limited,” a clear sign that this market lacks competition, most likely due to a lack of incentives for the private sector to invest in this class of devices.
Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).
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