Biologics
FDA has posted a draft guidance entitled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.” The document is intended to provide industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during cell/gene therapy development. The FAQs cover multiple disciplines, including regulatory review,
Drugs
FDA has accepted for review an Aldeyra Therapeutics NDA for reproxalap, a first-in-class investigational topical new drug candidate for treating signs and symptoms of dry eye disease. The agency has set a user fee review action target date of 4/2/2025. Reproxalap is described as a small-
FDA Policy/General
Federal Register notice: FDA is issuing an order permanently debarring Ivette Maria Portela Martinez from providing services to a person that has an approved or pending drug product application. The order is based on her felony conviction arising from drug development work at AMB Research Center (Miami, FL) where she
FDA Policy/General
Federal Register notice: FDA has determined that Merck’s Hydrocortone (hydrocortisone sodium phosphate) injection, equivalent to 50 mg/mL, was not withdrawn due to safety or effectiveness reasons. The determination will allow the agency to approve ANDAs that reference the product if all other legal and regulatory requirements are met.
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
Devices
FDA has issued an alert about safety concerns with the use of Getinge/Maquet’s VasoView HemoPro Endoscopic Vessel Harvesting (EVH) Systems, and a supply concern for EVH devices. In September, the company sent customers an Urgent Medical Device Removal notice telling them to not use the devices because there
FDA Policy/General
Federal Register notice: FDA is seeking comments on an information collection revision entitled “Prescription Drug User Fee Program.” The agency is revising the collection to include its current commitment goals from the document “PDUFA Reauthorization Performance Goals and Procedures Fiscal Years 2023 Through 2027. The commitment goals represent agreements reached
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
FDA Policy/General
President-elect Donald Trump’s pick as HHS secretary Robert F. Kennedy Jr. is promising to “free the agencies,” including FDA, “from the smothering cloud of corporate capture so they can pursue their mission to make Americans once again the healthiest people on Earth. In a 11/15 statement delivered
Biologics
FDA has accepted for review a Sanofi and Regeneron supplemental BLA resubmission for Dupixent (dupilumab) to treat adults and pediatric patients aged 12 years and older with chronic spontaneous urticaria (CSU) whose disease is not adequately controlled with H1 antihistamine treatment. The user fee review action target date is 4/
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
FDA Policy/General
President-elect Donald Trump has announced that he will nominate controversial FDA critic and vaccine skeptic Robert F. Kennedy Jr. as his secretary for Health and Human Services. “For too long, Americans have been crushed by the industrial food complex and drug companies who have engaged in deception, misinformation, and
FDA Policy/General
Federal Register notice: FDA is making available a draft guidance entitled “Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics.” The document provides recommendations on approaches for the nonclinical safety evaluation of oligonucleotide-based therapeutics (ONTs) to support clinical development and their marketing. The agency notes that ONTs “present unique challenges and
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
Devices
Since 2015, CDRH and CBER have granted 1,041 Breakthrough Device designations, with CDRH granting 1,029 designations and CBER granting 12. The totals include devices originally designated under the former Expedited Access Pathway, according to the FDA Breakthrough Device Program Web page. There were 206 devices designated in Fiscal
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’
Devices
FDA has granted a de novo marketing authorization to Omron Healthcare for its new home blood pressure monitors featuring artificial intelligence (AI)-powered atrial fibrillation detection. The company says its novel machine learning IntelliSense AFib algorithm automatically analyzes the pressure pulse wave generated during blood pressure measurement to detect AFib,
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
Devices
FDA has granted SonoMotion a de novo marketing authorization for its Stone Clear device for the anesthesia-free treatment of post-lithotripsy kidney stone fragments. The device is designed to deliver ultrasound pulses externally to move and help the passage of residual fragments without surgery, according to the company. The