Drugs
A 3/18-3/22 FDA inspection at the LCC Ltd. drug manufacturing facility in Chungcheongbuk-do, South Korea, found significant violations of current good manufacturing practice (CGMP) regulations and unapproved new drug violations. An 11/21 Warning Letter says specific CGMP violations were: * failure to test samples of each
Drugs
FDA has granted Merck a breakthrough therapy designation for sacituzumab tirumotecan (sac-TMT) and its use in treating patients with advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) mutations (exon 19 deletion [19del] or exon 21 L858R) whose disease progressed on or
Biologics
The House Select Subcommittee on the Coronavirus Pandemic has issued its final 550-page report that generally praises actions taken under the Trump administration and criticizes actions taken by the Biden administration. A committee statement (including a link to the full document) says “After Action Review of the Covid-19
Drugs
An FDA 4/29-5/3 Bioresearch Monitoring Program inspection at Applied Therapeutics in New York, NY, found that the firm did not adhere to the applicable statutory requirements and regulations governing the conduct of clinical investigations. The findings are presumably related to FDA’s recent action to issue a
Drugs
FDA has accepted for review a Cytokinetics NDA for aficamten, a cardiac myosin inhibitor for treating obstructive hypertrophic cardiomyopathy (HCM). The agency set a user fee review action target date of 9/26/2025. The submission is based on data from the Phase 3 SEQUOIA-HCM pivotal trial, which demonstrated
Drugs
Johnson & Johnson has submitted two supplemental BLAs for Tremfya (guselkumab) for treating children six years and older with moderate-to-severe plaque psoriasis (PsO) and children five years of age and older with active juvenile psoriatic arthritis (jPsA). Tremfya is currently approved for treating adults with moderate to severe
Drugs
FDA has issued Applied Therapeutics a complete response letter on its NDA for govorestat, a central nervous system-penetrant aldose reductase inhibitor (ARI) for treating classic galactosemia. The letter cited “deficiencies in the clinical application,” the company says, adding that it is requesting a meeting with the agency to discuss
Drugs
FDA has published a guidance, Use of Circulating Tumor DNA for Curative-Intent Solid Tumor Drug Development, to help sponsors planning to use circulating cell-free plasma-derived tumor DNA (ctDNA) as a biomarker in cancer clinical trials conducted under an IND and/or to support marketing approval for drugs
Drugs
FDA has released the form FDA-483 issued following a 5/18-5/26/2023 inspection at the Patheon Italia sterile drug manufacturing facility in Monza, Italy. The four inspection observations were: * aseptic processing areas are deficient regarding the system for monitoring environmental conditions; * failure to establish, write, and follow
Drugs
FDA has released a list of eight priority areas for the GDUFA science and research program in FY 2025. “These eight areas encompass scientific challenges that the generic industry and FDA’s generic drug program identify as being significant over the coming years, and they also represent opportunities for scientific
Drugs
PTC Therapeutics says the Phase 2 CardinALS study of utreloxastat failed to meet its primary endpoint of slowing ALS disease progression. A company statement says that while utreloxastat was demonstrated in the study to be safe and well tolerated, further development is not planned due to the lack of efficacy
Drugs
FDA says it has received serious adverse event reports associated with Bluebird Bio’s Skysona (elivaldogene autotemcel). A just-posted safety alert says that since its approval in April the agency has received reports about hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia. The autologous
Drugs
FDA has extended by three months its review of a Soleno Therapeutics NDA for DCCR (diazoxide choline) extended-release tablets for treating Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The new user fee review action target date is 3/27/2025. The agency says
Drugs
A 5/20-5/24 FDA inspection at Eurosirel S.P.A. in Milan, Italy, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 11/20 Warning Letter says the specific violations were: * failure to conduct at least one test to verify the identity of
Drugs
FDA has published a draft guidance, Assessment of Ovarian Toxicity in Premenopausal Adults During Drug Development for Oncologic Products, with recommendations to sponsors on measuring ovarian toxicity using clinical measures and biomarkers of ovarian function in relevant cancer clinical trials that enroll premenopausal women. The document says it specifically applies
Drugs
FDA has accepted for review a Satsuma Pharmaceuticals NDA resubmission for STS101 (dihydroergotamine nasal powder) for the acute treatment of migraine with or without aura. In January, FDA issued Satsuma a complete response letter on the original NDA due to chemistry, manufacturing, and controls considerations, according to the company. The
Drugs
Axsome Therapeutics says it is planning to submit an NDA based on just-reported Phase 3 trial data on AXS-12 (reboxetine), described as a highly selective and potent norepinephrine reuptake inhibitor and cortical dopamine modulator, for treating narcolepsy. The data showed that AXS-12 achieved the primary endpoint in
Drugs
FDA has posted a draft guidance entitled “Recommended Follow-Up Testing for an Ames-Positive Drug (Active Ingredient) or Metabolite To Support First-in-Human Clinical Trials With Healthy Subjects.” The document provides agency feedback on how CDER views positive findings in an in vitro bacterial reverse mutation (Ames) test
Drugs
FDA has released the form FDA-483 issued following a 3/6-3/14/2023 inspection at the Regeneron Ireland Designated Activity Company bulk drug substance manufacturing facility in Limerick, Ireland. The six inspection observations were: * failure of the quality unit to ensure issuance of documents is controlled and original
Drugs
FDA has issued a final guidance entitled “Transitional Enforcement Policy for Ethylene Oxide Sterilization Facility Changes for Class 3 Devices.” The document articulates the agency’s stance on expected EtO changes pursuant to new Environmental Protection Agency (EPA) requirements. The guidance provides recommendations for manufacturers whose devices are sterilized by
Drugs
FDA has accepted for review an Alnylam Pharmaceuticals supplemental NDA for vutrisiran, an investigational RNAi therapeutic in development for treating transthyretin amyloidosis (ATTR) with cardiomyopathy (ATTR-CM). The submission included a priority review voucher and the agency has set a user fee review action date of 3/23/2025. Vutrisiran
Drugs
FDA has approved a BridgeBio Pharma NDA for Attruby (acoramidis), a near-complete stabilizer of transthyretin for treating adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. The approval is based on data from the company’s ATTRibute-CM Phase 3 study that