Drugs

Disc Medicine Plans NDA Refiling After Favorable Trial Data

Disc Medicine says it will resubmit an NDA by year-end for bitopertin in adults and adolescents with erythropoietic protoporphyria (EPP) following just-reported APOLLO Phase 3 favorable data. According to the company, the results demonstrated statistically significant and clinically meaningful benefits across all study endpoints.

Earlier this year, FDA issued Disc a complete response letter (CRL) for its NDA seeking accelerated approval under the Commissioner’s National Priority Voucher pilot program. The letter said FDA had concluded that while mid-stage trial data showed the drug significantly reduced a key biomarker associated with the rare blood disorder, the application did not adequately demonstrate that biomarker changes were reasonably likely to predict clinical benefit, according to the company.

In June, Disc said it had reached agreement with the agency on a potential regulatory path forward for bitopertin. The agency indicated that a successful Phase 3 (APOLLO) trial could support a response to the drug's recent CRL and potentially lead to traditional approval for treating the rare genetic disorder characterized by severe, painful reactions to sunlight.

FDA Warns Baxter Over GMP Issues at Injectable Drug Plant

FDA has issued a warning letter to Baxter International citing significant GMP deficiencies at its Round Lake, IL, manufacturing facility, including recurring cross-contamination involving cephalosporin antibiotics, inadequate sterile manufacturing controls, particulate contamination and failures to properly investigate sterility test results. The just-posted 9/29 Warning Letter, which followed

Senators Urge FDA to Crack Down on ‘Research Use Only’ Drugs

Sen. Jim Banks (R-IN) is leading a group of senators urging FDA to ramp up enforcement against online vendors using “for research use only” labeling to market unapproved drugs and active pharmaceutical ingredients (APIs) to consumers, citing growing concerns about counterfeit versions of Eli Lilly’s investigational weight-loss

FDA Guidance on Drug-Related Software, Labeling Requirements

FDA has finalized a guidance entitled Regulatory Considerations for Prescription Drug Use-Related Software that outlines how prescription drug manufacturers should address software applications used with their products, including when claims about the clinical benefits of such software can be included in FDA-approved drug labeling. The guidance addresses mobile

Device QMSR Fears Easing: Panel

Industry executives and FDA officials who participated in a 10/19 panel at The MedTech Conference say initial industry concerns about the new Quality Management System Regulation (QMSR) and its implementation by FDA are easing as companies get more experience with inspections under the new protocol. A Regulatory Affairs Professionals

Read more

FDA Warns Baxter Over GMP Issues at Injectable Drug Plant

Senators Urge FDA to Crack Down on ‘Research Use Only’ Drugs

FDA Guidance on Drug-Related Software, Labeling Requirements

Device QMSR Fears Easing: Panel