Drugs
CorMedix says that following a meeting with FDA, it plans to resubmit its NDA for DefenCath by mid-May. A company statement says that at the meeting, FDA indicated it had received the close-out report for inspectional observations at the CorMedix contract manufacturing organization and the NDA could be
Drugs
Harvard Medical School researchers say real-world evidence studies can reach similar conclusions as randomized clinical trials (RCT) when design and measurements can be closely emulated, although such emulation may be difficult to achieve. Writing in the Journal of the American Medical Association, the researchers say they emulated the design
Drugs
With positive results from the Phase 3 APPOINT-PNH trial showing that the investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients, Novartis says it is planning global regulatory submissions, including FDA, in the first half of this year. The company
FDA Policy/General
Federal Register notice: FDA is seeking comments on an information collection extension “Animal Drug and Animal Generic Drug User Fee Programs.” The collection supports FDA activities to operate the user fee program. To view the notice, click here.
Drugs
FDA has released the form FDA-483 issued following a 3/6-3/17 inspection at Alvotech Hf, a Reykjavik, Iceland, drug substance and drug product manufacturer. The eight inspection observations were: * failing to have in writing and fully follow the responsibilities and procedures applicable to the quality unit; * failing
Drugs
Akorn (Gurnee, IL) through its bankruptcy (Chapter 7) trustee is recalling various within-expiry human and animal products because the company has ceased all operations, including quality activities. In February, Akorn announced that it failed to find a buyer for its operations and terminated all employees (see letter). “The discontinuation
Drugs
FDA has approved a Vertex Pharmaceuticals supplemental NDA for the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor) to include children with cystic fibrosis (CF) aged two through five years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the
FDA Policy/General
FDA has issued a warning about adverse event reports related to selective androgen receptor modulators (SARMs), which are often marketed as dietary supplements but the agency considers them unapproved drugs. SARMs are unapproved chemical substances that “mimic the effects of testosterone and anabolic steroids,” the agency says, warning that online
Drugs
FDA is asking the Oncologic Drugs Advisory Committee (ODAC) to discuss whether an sNDA for AstraZeneca’s olaparib (Lynparza) should be restricted to selected patients. An agency briefing document released in advance of the 4/28 ODAC meeting says AZ is seeking a broad indication for olaparib in combination with
Devices
FDA has lifted a 2021 Warning Letter to Medtronic that followed an inspection of the company’s diabetes headquarters in Northridge, CA. “The resolution of the Warning Letter follows ongoing remediation actions from the company and proactive actions to continue to strengthen its quality systems,” the company says. “All regulatory
Drugs
An 11/6-11/10/22 FDA inspection at the Pharmaplast drug manufacturing facility in Alexandria, Egypt, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 4/13 Warning Letter says the specific violations were: * failing to test samples of each component for identity and
FDA Policy/General
Florida has filed a second suit against FDA charging the agency is violating the Freedom of Information Act (FOIA) by withholding records requested by the state relative to its application to permit the importation of Canadian drugs. The 4/21 suit says FDA violated the FOIA by not properly responding
Devices
An 8/8-8/11/22 FDA inspection at Medivance Instruments in London, UK, determined that the firm manufactures the Velopex Aquacut Fluid Abrasion Unit and accessories as part of its Velopex AquaCare dental abrasion line of products. A just-released 2/13 Warning Letter says the firm holds a
Drugs
FDA says its April review of Web sites maintained by Medolife Rx in Burbank, CA, determined that it markets several unapproved new drugs. A 4/19 Warning Letter says the products are concerning from a public health perspective because they claim to cure, mitigate, treat, or prevent serious and/or
Drugs
FDA Division of Oncology director Harpreet Singh says efforts to streamline oncology clinical trials can produce discomfort in investigators over issues such as safety reporting being not as rigorous as investigators expect FDA to want. Singh was a panelist on an American Society of Clinical Oncology podcast, discussing FDA’s
Drugs
FDA has granted Biogen accelerated approval for Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutation, which in turn is linked with 20% of familial cases and about 2% of sporadic ALS cases. The antisense oligonucleotide, which is designed to target
Drugs
FDA has lifted a clinical hold against a MaaT Pharma IND for an open-label, single arm Phase 3 pivotal clinical trial evaluating the safety and efficacy of MaaT013 to treat gastrointestinal acute Graft-versus-Host Disease as a third line treatment. “This is the first time the agency has
Drugs
Senate Health, Education, Labor, and Pensions (HELP) Committee chairman Bernie Sanders (I-VT) and ranking member Bill Cassidy (R-LA) announced 4/25 that a legislative bipartisan deal has been reached to expand the availability of low-cost generic drugs, clarify orphan drug exclusivity, and reform the pharmacy benefit managers
FDA Policy/General
Federal Register notice: FDA is seeking comments on an information collection extension entitled “Customer/Partner Service Satisfaction Surveys.” Agency regulations give it authority to conduct research and public information programs about regulated products and its responsibilities. The collection also supports Executive Order 12862, “Setting Customer Service Standard,” that directs federal
Drugs
FDA has sent a letter to Sun Pharma telling the company that it is out of compliance with a 2012 consent decree after an inspection last year at the company’s Mohali (Punjab), India cited significant GMP violations and the agency classified the inspection as “Official Action Indicated." “U.
Drugs
The U.S. Supreme Court late 4/21 granted FDA a stay of Texas federal judge Matthew Kacsmaryk’s nationwide ban on mifepristone while it is appealed in the U.S. Court of Appeals for the Fifth Circuit. Oral arguments are on fast track in that venue and are scheduled
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) is planning a survey to better understand quantitative claims in direct-to-consumer prescription drug advertising. The office notes that DTC advertising does include quantitative claims about a drug’s efficacy or risks and there is existing guidance, “Presenting Quantitative Efficacy and