Regulatory

Liquidia Corporation said that the FDA has accepted its sNDA to add pulmonary hypertension associated with interstitial lung disease (PH-ILD) as an indication for Yutrepia treprostinil inhalation powder without requiring any additional studies and has set the PDUFA goal date as January 24, 2024. The FDA tentatively approved Yutrepia for the treatment of … [Read more...] about FDA sets PDUFA date for Liquidia’s sNDA to add PH-ILD indication for Yutrepia treprostinil DPI

According to ARS Pharmaceuticals, the FDA has issued a complete response letter to the company's NDA for Neffy epinephrine nasal spray for the treatment of allergic reactions, including anaphylaxis, and the agency has requested the completion of a PK/PD repeated-dose study prior to approval. The CRL also asked for more information on nitrosamine impurities, which the … [Read more...] about FDA issues CRL to ARS’ NDA for Neffy intranasal epinephrine for the treatment of allergic reactions

Orexo announced that it has resubmitted an NDA for OX124 naloxone nasal powder for the reversal of opioid overdose. In February 2023, the company announced that it had submitted an NDA for OX124 and then in April announced that it would have to resubmit the application due to "unexpected technical issues with the equipment used for the secondary packaging process in … [Read more...] about Orexo resubmits NDA for OX124 intranasal naloxone

The FDA issued a document on September 8, 2023 titled "Application of Human Factors Engineering Principles for Combination Products: Questions and Answers," which is the final version of a draft guidance titled "Human Factors Studies and Related Clinical Study Considerations in Combination Product Design and Development" that was issued in 2016. According to the … [Read more...] about FDA issues final guidance on human factors principles for combination products

NurExone Biologic announced that the company has received feedback from the FDA following a pre-IND meeting regarding NurExone's ExoPTEN intranasal exosome therapy and now plans to submit an IND by the end of this year with the goal of initiating Phase 1/2 clinical trials in 2025. In July 2022, the company said that it had contracted with Polyrizon for development of … [Read more...] about NurExone says that it held pre-IND meeting with the FDA regarding ExoPTEN intranasal therapy for spinal cord injuries

According to Verona Pharma, the FDA has accepted the company's NDA for its nebulized ensifentrine (RPL554) for the treatment of COPD with a PDUFA target date of June 26, 2024. The company submitted the NDA for ensifentrine, a PDE3/PDE4 inhibitor, at the end of June 2023. Verona is also developing DPI and MDI formulations of ensifentrine. Verona … [Read more...] about FDA accepts Verona’s NDA for nebulized ensifentrine for the treatment of COPD

According to Krystal Biotech, the FDA has granted orphan drug designation to the company's KB408 nebulized gene therapy for the treatment of alpha-1 antitrypsin (AAT) deficiency. Krystal, which is also developing inhaled gene therapies for the treatment of cystic fibrosis (KB407) and solid tumors (KB707), says that KB408 is intended to deliver 2 copies of the … [Read more...] about Krystal Biotech gets orphan drug designation for KB408 inhaled gene therapy for AAT deficiency

The FDA issued a warning letter to AstraZeneca dated August 4, 2023 that cites multiple "false or misleading claims and/or representations" about the efficacy of the company's Breztri Aerosphere (PT010) budesonide / glycopyrrolate / formoterol fumarate MDI. Breztri was approved by the FDA for the treatment of COPD in July 2020. According to the warning letter, the … [Read more...] about FDA issues warning letter to AstraZeneca over efficacy claims for Breztri MDI