Drugs
Ionis Pharmaceuticals says data from its Phase 1/2 open-label study of ION582 in people with Angelman syndrome demonstrated consistent and encouraging clinical improvement on measures assessing all “functional domains” including communication, cognition and motor function. “Overall, 97% of people in the medium and high dose groups saw an
Drugs
FDA has cleared a Varian 510(k) for new functionality to its Ethos radiotherapy system for treating cancer. The latest version of the device introduces a streamlined workflow for daily treatment adaptation across more areas of the body, and provides greater connectivity to patient data to make it easier to
Drugs
FDA has posted a draft guidance entitled “Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products; Questions and Answers.” The document provides answers to common questions from applicants and other interested parties about postapproval manufacturing made to licensed biosimilars and interchangeable biosimilars. It is intended to inform applicants about the
Drugs
FDA has granted Diamyd Medical a Fast Track designation for Diamyd (rhGAD65/alum) for use in treating Type 1 diabetes in pediatric patients with Stage 1 or Stage 2 Type 1 diabetes carrying the genotype HLA DR3-DQ2. Earlier this year, Diamyd was granted fast track for treating Stage 3
Drugs
FDA has granted Neuren Pharmaceuticals a rare pediatric disease designation for its drug candidate NNZ-2591 and its use in treating Phelan-McDermid syndrome. The designation may qualify Neuren for a priority review voucher if its therapy gains approval. The designation is based on recent data from a Phase 2
Drugs
During an end-of-Phase 2 meeting with Agenus on its immunotherapy combination botensilimab (BOT) and balstilimab (BAL) to treat adults with relapsed/refractory microsatellite stable colorectal cancer with no liver metastases, FDA advised the company not to submit its trial results for accelerated approval, saying that objective response rates
Drugs
FDA has classified as “official action indicated” an April inspection of Zydus Lifesciences’ injectables manufacturing facility at Jarod, Gujarat, India. The company says it will “work closely with the agency to resolve the regulatory status of this facility expeditiously.” The inspection resulted in a 10-item Form FDA-483 that
Drugs
Aveo Oncology says its TiNivo-2 Phase 3 clinical trial in patients with advanced metastatic renal cell carcinoma whose tumors had progressed following prior immune checkpoint inhibitor treatment did not meet the primary endpoint of increasing progression-free survival when nivolumab (Bristol Myers Squibb’s Opdivo) was added to low-
Drugs
FDA has approved a Phathom Pharmaceuticals NDA for Voquezna (vonoprazan) 10 mg tablets for relieving heartburn associated with non-erosive gastroesophageal reflux disease (non-erosive GERD) in adults. This is the third approval for Voquezna, having seen previous approvals for treating all severities of erosive esophagitis, and combination use with
Drugs
FDA has published a guidance, Clinical Pharmacology Considerations for Human Radiolabeled Mass Balance Studies, with agency recommendations for such studies in investigational drugs, including (1) deciding whether and when to conduct the study, (2) designing the study, and (3) reporting results. The document’s recommendations are based, in part, on
Drugs
FDA has released the form FDA-483 issued following a 3/20-4/5 inspection at Denver Solutions, an Englewood, CO, outsourcing facility doing business as Leiters Health. The seven inspection observations were: * failure to thoroughly review any unexplained discrepancy and the failure of a batch or any of its
Drugs
CDER director Patrizia Cavazzoni and CBER director Peter Marks say they will co-lead a new Rare Disease Innovation Hub (the Hub) to work across rare diseases, but to especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.
Drugs
Endo is recalling (Class 1) one lot of clonazepam orally disintegrating tablets 0.25 mg due to mislabeling on the cartons of some packs that shows the product strength as 0.125 mg and not 0.25 mg. The blister strips inside the product pack reflect the correct strength of
Drugs
Based on positive results from the Phase 3 ARANOTE trial of its Nubeqa (darolutamide) in men with metastatic hormone-sensitive prostate cancer, Bayer says it will discuss with FDA a regulatory submission to expand the drug’s indication. A company statement says the trial of Nubeqa with androgen deprivation therapy
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
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
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