Drugs
FDA has granted Sellas Life Sciences Group a Rare Pediatric Disease Designation (RPDD) for SLS009, a highly selective CDK9 inhibitor for treating pediatric acute myeloid leukemia (AML). Previously, the agency granted Orphan Drug and Fast Track Designations for the therapy. The company says it is continuing enrollment in a pediatric
FDA Policy/General
Federal Register notice: FDA is announcing that Argenx’s supplemental NDA for Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), indicated for treating chronic inflammatory demyelinating polyneuropathy, was approved 6/21 using a priority review voucher. Previously, the drug was approved for treating generalized myasthenia gravis. To view the notice, click
Drugs
FDA says its review of records submitted by YangZhou SuXiang Medical Instrument Co., Ltd., in Yangzhou City, China, in response to an agency request, identified significant violations of current good manufacturing practice (CGMP) regulations in its manufacturing of finished drugs. A 7/8 Warning Letter says specific violations were: * failure
Drugs
A 1/8-1/16 FDA inspection at Jiangsu Hengrui Pharmaceuticals in Lianyungang, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 7/11 Warning Letter says specific violations were: * failure of the firm’s quality unit to exercise its responsibility to ensure drug
Biologics
FDA has published a guidance, Application User Fees for Combination Products, explaining how fees are assigned for combination products reviewed in a single application or separate applications for each component. The document says the guidance replaces one issued in 4/2005 to provide consistency with current user fee programs and
Drugs
FDA has accepted for review a resubmitted Lexicon Pharmaceuticals NDA for Zynquista (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with Type 1 diabetes and chronic kidney disease (CKD). The agency indicated that it considers the resubmission to be a complete response to its 2019 complete
Drugs
FDA has published a draft guidance, Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment, to help sponsors in the clinical development of drugs to treat pediatric patients with inflammatory bowel disease (IBD). The document says the draft specifically provides agency recommendations about the necessary attributes of clinical studies for drugs
Drugs
FDA commissioner Robert Califf says he hopes the agency will look for opportunities to enable innovation in stalled areas in chronic disease treatment. Writing in an FDA Voices online post, Califf reviews statistics showing that seven of the 10 leading causes of death in the U.S. are common chronic
Devices
FDA has cleared a Smith+Nephew 510(k) for its new Catalystem Primary Hip System, which the company says is designed to address the increased adoption of anterior approach procedures and the expanding role of ambulatory surgery centers. “Advancements in primary hip arthroplasty include a shift to a shorter, proximally
Biologics
CBER says a Bioresearch Monitoring Program remote regulatory assessment of the Massachusetts Institute of Technology Committee on the Use of Humans as Experimental Subjects institutional review board (IRB) conducted between 5/30 and 6/9/2023 found “objectionable conditions” that were repeated observations from previous reviews. A just-released 6/
Drugs
FDA has given DiscGenics the green light for a Phase 3 clinical trial after agreeing to the clinical protocols and chemistry, manufacturing, and controls clinical development plan for its allogeneic injectable disc progenitor cell therapy (IDCT or rebonuputemcel) for painful lumbar degenerative disc disease. “There are very few credible treatments
Drugs
FDA has issued Orexo AB a complete response letter on its NDA for OX124, a high-dose naloxone rescue medication being developed for opioid overdose. The letter cites the need for an additional human factors (HF) study, and requests additional technical data on the final commercial product, according to the
Drugs
FDA has released a guidance entitled “Drugs for Treatment of Partial Onset Seizures: Extrapolation of Efficacy from Adults to Pediatric Patients One Month of Age and Older.” The document provides recommendations to sponsors on the clinical development of drugs for the treating partial onset seizures (POS) in pediatric patients. Specifically,
Drugs
Lexeo Therapeutics says it will explore with FDA the potential to seek accelerated approval for LX2006 and its use in treating Friedreich ataxia (FA) cardiomyopathy based on just-announced “positive interim data” from two early-stage trials. “Across both the Lexeo SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and
Drugs
Pharmaceutical marketer Quintan Cockerell (Palos Verdes Estates, CA) was sentenced 7/12 to two years and five months in prison and ordered to pay over $59 million in restitution for conspiring to defraud the U.S., receiving illegal kickbacks in exchange for compounded medications prescription referrals, and money laundering. According
Devices
CDRH is announcing new organizational changes to help the Center meet commitments under the Medical Device User Fee Amendments reauthorization and other strategic priorities. For example, CDRH is elevating the Office of Communication and Education into a super office called “Office of Communication, Information Disclosure, Training, and Education (OCITE)” and
Devices
Baxter is recalling (Class 1) its Life200 Ventilator System due to the potential for battery failure involving its charging dongle, which can prevent the system from charging or only allow for intermittent charging. “Use of affected products may cause serious adverse health consequences, including a drop in oxygen levels (desaturation
Drugs
A 3/2023 inspection at a Lonza Visp, Switzerland manufacturing facility has resulted in a one-item Form FDA-483. The inspection found that the company’s laboratory records do not include complete data derived from all tests, examinations and assays necessary to assure compliance with established specifications and standards,
FDA Policy/General
Federal Register notice: FDA is announcing the Cellular and Gene Therapies Interactive Site Tours Program that is intended to give CBER regulatory project managers and reviewers an opportunity to tour biotechnology manufacturing facilities developing cellular and gene therapy products. The program also allows the participants to exchange regulatory experiences with
Drugs
FDA has granted Sumitomo Pharma America a fast track designation for DSP-5336 and its use in treating patients with relapsed or refractory acute myeloid leukemia with a KMT2A rearrangement, also known as mixed lineage leukemia rearrangement or nucleophosmin mutation. DSP-5336 is described as a small molecule inhibitor of
Devices
Former FDA commissioner Scott Gottlieb (2017-2019) says Congress needs to design a framework for the modern, efficient regulation of medical artificial intelligence that could be based on the regulatory scheme articulated in the pending Verifying Accurate Leading-edge IVCT Development Act (VALID Act — bills S 2209 and HR 4128)
Drugs
Indivior says it is discontinuing sales and marketing of schizophrenia drug Perseris (risperidone) due to “expected adverse impacts from increased payor management of the category that crystalized in [the second quarter] and that are expected to make the product no longer financially viable.” The once-monthly subcutaneous risperidone-containing, long-