Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older.
The draft Medicare physician fee schedule for calendar year 2025 provoked a strong backlash from physician societies due to a net cut in physician pay. Device makers have their own concerns, including the latest in a series of controversies over coverage of skin substitutes.
In what represents its first patenting, Roseville, Minn.-based Iveacare Inc. provides insights as to what its first therapeutic target will likely be since the developer of neuromodulation therapy devices emerged from stealth mode in April 2024 with the closing of $27.5 million series A financing.
The U.S. Court of Appeals for the Federal Circuit reined in a district court that invalidated three claims in an Astellas Pharma Inc. patent protecting bladder drug Myrbetriq (mirabegron) based on a issue that was never argued.
What comes down must go up seems to be the rule of thumb these days, even for U.S. FDA fees. The cost of using a priority review voucher (PRV) to shave four months off an FDA standard 10-month drug review has dropped drastically over the past several years. But come Oct. 1, biopharma companies will have to pay nearly 89% more than on the previous day to use a PRV.
Clearly frustrated by the U.S. FDA’s rejection of tradipitant to treat gastroparesis, Vanda Pharmaceuticals Inc. CEO Mihael Polymeropoulos said the agency is discouraging drug development for the serious gastric emptying condition for which there has been no new treatment in 40 years. The complete response letter (CRL) issued on the Sept. 18 PDUFA date for the neurokinin-1 receptor antagonist, which Vanda licensed from Eli Lilly and Co. in 2012, requested additional studies that the company said are not in line with the advice of experts in the field.
After disclosing data from the phase II Herald study, Aligos Therapeutics Inc. may sign a partner to help advance ALG-055009, a thyroid hormone receptor (THR) beta agonist, in subjects with metabolic dysfunction-associated steatohepatitis (MASH, formerly nonalcoholic steatohepatitis, or NASH).
Positive data from two studies boosted Edgewise Therapeutics Inc.’s market share and elevated analyst enthusiasm for the company and its treatment of obstructive hypertrophic cardiomyopathy. Top-line data from the phase I and phase II studies of EDG-7500 in treating the genetic disease that results in thickened heart muscles showed the therapy was well-tolerated in healthy volunteers and produced meaningful improvements in those with the disease.
Avicenna.ai SAS received U.S. FDA clearance for its Cina-Cspine algorithm, which uses machine learning technology to detect and triage cervical spine fractures from computed tomography images. The tool automatically flags imaging findings that are compatible with acute cervical spine fractures, which can cause serious neurological damage or paralysis.
Scientists and doctors are getting much better at developing treatments for cancer. More than 18 million people with a history of cancer were alive in the U.S. at the beginning of 2022, up from just 3 million in 1971, according to the 14th edition of the American Association for Cancer Research (AACR) Cancer Progress Report. And that number is expected to rise to 26 million by 2040.
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