Aculys Pharma says that it plans to move ahead with submission of a marketing application to Japanese authorities based on interim data showing that a Japanese Phase 3 clinical study of NRL-1 diazepam nasal spray in epilepsy patients aged 6 to 17 met its primary efficacy endpoint. Aculys acquired development and commercialization rights to Neurelis's NRL-1 diazepam … [Read more...] about Aculys says it will file NDA based on interim analysis of Japanese Phase 3 trial of NRL-1 (Valtoco) diazepam nasal spray
Regulatory
FDA places clinical hold on GH Research IND for GH001 inhaled 5-MeO-DMTFDA
GH Research announced that the FDA has placed a clinical hold on an IND that the company submitted for GH001 inhaled mebufotenin (5-MeO-DMT) for the treatment of treatment-resistant depression, citing "Insufficient information to assess risks to human subjects." The company said that it is waiting to learn more details from the FDA about the reasons for the clinical … [Read more...] about FDA places clinical hold on GH Research IND for GH001 inhaled 5-MeO-DMTFDA
FDA accepts Defender’s NDA for DPI-386 scopolamine nasal gel for priority review
According to Defender Pharmaceuticals, the FDA has accepted the company’s NDA for DPI-386 scopolamine nasal gel for the treatment of motion sickness for priority review, with a PDUFA goal date of January 26, 2024. Defender submitted the NDA in July 2023. The company said that the FDA granted priority review at the request of the US Navy. Defender President and CEO … [Read more...] about FDA accepts Defender’s NDA for DPI-386 scopolamine nasal gel for priority review
FDA sets PDUFA date for Liquidia’s sNDA to add PH-ILD indication for Yutrepia treprostinil DPI
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
Krystal Biotech announces plans for Phase 1 trial of KB408 inhaled gene therapy for the treatment of AAT
Krystal Biotech said that the FDA has cleared an IND for KB408 nebulized gene therapy for the treatment of alpha-1 antitrypsin deficiency, and the company said that it plans to initiate a Phase 1 trial in early 2024. According to Krystal, the study will evaluate 3 ascending doses of KB408 in adult patients with AAT deficiency who have a PI*ZZ genotype. KB408 is … [Read more...] about Krystal Biotech announces plans for Phase 1 trial of KB408 inhaled gene therapy for the treatment of AAT
FDA issues CRL to ARS’ NDA for Neffy intranasal epinephrine for the treatment of allergic reactions
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 resubmits NDA for OX124 intranasal naloxone
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
FDA issues final guidance on human factors principles for combination products
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 says that it held pre-IND meeting with the FDA regarding ExoPTEN intranasal therapy for spinal cord injuries
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
FDA accepts Verona’s NDA for nebulized ensifentrine for the treatment of COPD
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