NurExone Biologic announced that the EMA has granted orphan medicinal product designation to NurExone's ExoPTEN intranasal exosome therapy, which the company is developing for the treatment of spinal cord injury. In 2023, the FDA granted orphan drug designation to ExoPTEN, and the company held a pre-IND meeting with the agency in anticipation of beginning clinical … [Read more...] about NurExone’s ExoPTEN intranasal exosome therapy for spinal cord injuries get orphan designation from the EMA
Regulatory
AlveoGene’s AVG-002 inhaled gene therapy gets rare pediatric disease designation for SP-B deficiency
UK-based start-up AlveoGene announced that its AVG-002 nebulized gene therapy for neonatal surfactant protein B (SP-B) deficiency has received a rare pediatric disease designation from the FDA. The company says that it will advance AVG-002 into clinical development and could submit a marketing application as soon as 2028. According to Alveogene, studies in SP-B … [Read more...] about AlveoGene’s AVG-002 inhaled gene therapy gets rare pediatric disease designation for SP-B deficiency
Satsuma resubmits its NDA for STS101 DHE nasal powder for migraine
Satsuma Pharmaceuticals announced that it has resubmitted its NDA for STS101 intranasal dry powder dihydroergotamine for the acute treatment of migraine with or without aura. The company initially submitted the NDA in March 2023 even though the Phase 3 SUMMIT trial of STS101 failed to meet its primary endpoints. In January 2024, the FDA issued a CRL to the … [Read more...] about Satsuma resubmits its NDA for STS101 DHE nasal powder for migraine
FDA clears Qnovia’s IND for Phase 1 study of QN-01 RespiRx inhaled nicotine
According to Qnovia (formerly Respira Technologies), the FDA has cleared an IND submitted by the company for a trial of its QN-01 RespiRx nicotine inhaler, which the company is developing for smoking cessation. The company says that it plans to initiate the Phase 1 study, which is expected to enroll 24 smokers, by the end of the year. The trial will compare nicotine … [Read more...] about FDA clears Qnovia’s IND for Phase 1 study of QN-01 RespiRx inhaled nicotine
FDA approves FluMist intranasal flu vaccine for self-administration
The FDA and AstraZeneca are both touting the agency's approval of FluMist intranasal influenza vaccine for caregiver administration to children aged 2-17 and for self-administration by adults up to age 49. The agency agreed to review AstraZeneca's sBLA for the self-administration change in October 2023. FluMist will still be available for healthcare provider … [Read more...] about FDA approves FluMist intranasal flu vaccine for self-administration
FDA warns agains use of Green Pharmaceutical’s SnoreStop nasal spray due to microbial contamination
The FDA has advised California-based Green Pharmaceuticals to recall its SnoreStop nasal spray due to "significant microbial contamination in SnoreStop Nasal Spray lot number 2460" detected during an inspection in April 2024 and is warning customers not to use the product. The FDA notes that the homeopathic product is unapproved and that Green previously recalled a … [Read more...] about FDA warns agains use of Green Pharmaceutical’s SnoreStop nasal spray due to microbial contamination
MannKind gets go-ahead for Phase 3 ICoN-1 trial of MNKD-101 clofazimine inhalation suspension in for NTM Japan
MannKind Corporation announced that the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) has cleared the initiation of the Phase 3 ICoN-1 trial of MNKD-101 clofazimine inhalation suspension for the treatment of nontuberculous mycobacterial (NTM) lung disease. The company noted that the trial has already been cleared in the US, South Korea, and Australia; and … [Read more...] about MannKind gets go-ahead for Phase 3 ICoN-1 trial of MNKD-101 clofazimine inhalation suspension in for NTM Japan
Silo Pharma says that it completed pre-IND meeting with the FDA regarding SPC-15 intranasal ketamine for PTSD and anxiety
Silo Pharma announced that it has received responses from the FDA following a pre-IND meeting regarding SPC-15 soft mist intranasal ketamine for post-traumatic stress disorder (PTSD) and stress-induced anxiety disorder. Silo is planning to take advantage of the 505(b)(2) pathway for SPC-15, which it has licensed from Columbia University. In November 2023, Silo … [Read more...] about Silo Pharma says that it completed pre-IND meeting with the FDA regarding SPC-15 intranasal ketamine for PTSD and anxiety
AstraZeneca says that it has completed studies to support regulatory submissions for LGWP propellant version of Breztri (Trixeo) Aerosphere MDI
AstraZeneca announced that it now has the data to support regulatory submissions for a new formulation of of Breztri (Trixeo) Aerosphere budesonide / glycopyrronium / formoterol using Honeywell's Solstice Air HFO 1234ze(e), a low global warming potential (LGWP) propellant. The company says that it now plans to initiate submissions for the reformulated MDI by the end … [Read more...] about AstraZeneca says that it has completed studies to support regulatory submissions for LGWP propellant version of Breztri (Trixeo) Aerosphere MDI
ARS Pharmaceuticals submits sNDA for Neffy 1 mg epinephrine nasal spray for anaphylaxis in small children
ARS Pharmaceuticals announced that it has submitted a supplemental NDA for Neffy 1 mg epinephrine nasal spray for the treatment of anaphylaxis in children weighing 15 to 30 kg (33-66 lbs). The FDA recently approved a 2 mg dose of Neffy for the treatment of anaphylaxis in patients weighing 30 kg or more. At that time, the company said that it intended to submit the … [Read more...] about ARS Pharmaceuticals submits sNDA for Neffy 1 mg epinephrine nasal spray for anaphylaxis in small children