Drugs
A new Government Accountability Office (GAO) report examining FDA’s programs for advancing rare disease products says training, collaboration with patient advocacy groups, and a multi-layered review process are key strategies the agency should rely on to maintain expertise and consistency in rare disease drug reviews. The report was
Drugs
A 10/2023 FDA inspection of Astellas Pharma’s Shizuoka, Japan manufacturing facility resulted in a just-posted one-item Form FDA-483. The agency investigators cited the facility’s complaint processing because its records were deficient. Specifically, the form said that a review of market complaints “revealed a lack
Drugs
Eyenovia says it is terminating the Phase 3 CHAPERONE study evaluating its proprietary drug-device combination of low-dose atropine in the company’s Optejet dispensing platform as a potential treatment for pediatric progressive myopia. The action is based on an independent data review committee analysis that found the trial
Drugs
Six weeks ahead of its user fee review action target date, FDA has approved a Syndax Pharmaceuticals NDA for Revuforj (revumenib), a menin inhibitor for treating relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients aged one year and older. Approval
Drugs
Philips Respironics has recalled (Class 1) its Garbin Evo ventilators due to the potential for aerosol deposit accumulation on the device’s internal flow sensor, which could lead to inaccurate flow measurements. An Urgent Medical Device Correction letter was recently mailed to consignees that provides instructions to users to prevent
Drugs
The Biotechnology Innovation Organization (BIO) is asking FDA to include more detailed strategic plans, complete with actionable deliverables and specific timelines, in the agency’s draft strategy document on innovative manufacturing technologies. “Specifically,” BIO says, “we recommend that the strategic plan and suggested activities outlined in the document include how
Drugs
FDA has approved an Atamyo Therapeutics IND for a Phase 1b/2b clinical trial involving ATA-200, a one-time gene therapy for treating γ-sarcoglycan-related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). The serious and debilitating condition is said to affect children and leads to loss
Drugs
FDA has approved PTC Therapeutics’ Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated to treat adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. An agency statement says Kebilidi is the first FDA-approved gene therapy to treat AADC deficiency. The therapy’
Drugs
The Sedgwick Recall Index says that for the third quarter of 2024, U.S. medical device recall events increased 8.3% from 242 in the second quarter to 262. The increase means that 2024 is on track to reach a three-year high, the Sedgwick report says. Device failure was
Drugs
FDA has released the form FDA-483 that was issued following a 6/17-7/2 inspection at the U.S. Specialty Formulations outsourcing facility in Allentown, PA. The seven inspection observations were: * the quality control unit lacks the responsibility and authority to approve and reject all components, drug product
Drugs
FDA has published a draft guidance, Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics, with recommendations on the approaches for the nonclinical safety evaluation of oligonucleotide-based therapeutics (ONTs) to support clinical development and marketing of those products. The document says although current guidance for small molecule drugs and therapeutic proteins
Biologics
Six Squire Patton Boggs attorneys say that at the end of the Supreme Court’s 2023-2024 term, the court issued four decisions that “have potentially wide-ranging consequences on federal agency rulemaking, adjudication, and regulatory compliance.” Writing in an online post, the attorneys analyze the impact of the decisions
Drugs
DifGen Pharmaceuticals is petitioning FDA to not approve any ANDA for Prednisolone Ophthalmic Solution 1% without the inclusion of long-term stability studies conducted under refrigerated conditions. The 11/7 petition specifically asks that the agency: * require generic applicants to conduct long-term stability studies that specifically demonstrate the product’
Drugs
Federal Register notice: FDA is announcing a 12/13 virtual public workshop entitled “Patient-Focused Drug Development: Workshop to Discuss Methodologic and Other Challenges Related to Patient Experience Data.” It is intended to highlight and discuss methodological issues related to patient experience data, including submitting and evaluating patient experience data
Drugs
Boston Scientific has recalled (Class 1 — use instructions update) its POLARx and POLARx FIT Cryoablation Balloon Catheters because of atrio-esophageal fistula (AE fistula) risks. AE fistula is a known and inherent risk for patients undergoing catheter ablation for atrial fibrillation, according to the company. “Although uncommon, esophageal injury is
Drugs
Speaking on a Government Accountability Office (GAO) podcast, GAO healthcare director Mary Denigan-Macauley says FDA needs to have a plan to address all of the causes that make it difficult to recruit facility inspectors and a strategy for retaining new hires. The podcast focuses on foreign and domestic facility
Drugs
An FDA review of records submitted at the agency’s request by the over-the-counter drug producer Zhejiang Qimei Cosmetics Co. in Zhejiang Sheng, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 11/7 Warning Letter says specific violations were: * failure of
Drugs
Federal Register notice: FDA has determined that Nuplazid (pimavanserin tartrate) tablet (equivalent 17 mg base) was not withdrawn due to safety or effectiveness concerns. The determination allows the agency to approve ANDAs that reference the product if all other legal and regulatory requirements are met. Nuplazid is an atypical antipsychotic
Drugs
The Republican leadership of the House Energy and Commerce Committee is asking the Government Accountability Office (GAO) to “examine the structure of laboratory safety programs” at FDA, the Centers for Disease Control (CDC), and the National Institutes of Health. An 11/12 committee statement (includes a link to a letter
Drugs
FDA has issued Intercept Pharmaceuticals a complete response letter on its supplemental NDA for Ocaliva (obeticholic acid) seeking to convert its accelerated approval to full approval for treating patients with primary biliary cholangitis (PBC). The company says that the letter was consistent with a September Gastrointestinal Drugs Advisory Committee meeting
Drugs
Eli Lilly says it opposes the request by the Outsourcing Facilities Association (OFA) that tirzepatide (Lilly’s Mounjaro and Zepbound) be added to the “clinical need” list, otherwise known as the 503 Bulks List. A Lilly comment says that OFA seeks permanent authorization for its members to engage in the
Drugs
Rapt Therapeutics says it is terminating its zelnecirnon (RPT193) drug development program due to a serious safety issue. Zelnecirnon was being evaluated in two randomized, placebo-controlled Phase 2 clinical trials in asthma and atopic dermatitis (AD), and both trials were placed on clinical hold by FDA earlier this year