Quote from Chief Pharmacist on October 4, 2024, 6:26 am

FDA Approves Aqneursa (levacetylleucine) to Treat Niemann-Pick Disease, Type C

“This is the second treatment the FDA has approved for NPC within the span of a week. Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases,” said Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “This approval again demonstrates the FDA’s commitment to work with the scientific community to overcome the unique challenges that may arise with rare disease drug development.”

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-new-drug-treat-niemann-pick-disease-type-c

Quote from Chief Pharmacist on October 5, 2024, 6:51 am

FDA Approves Nasal Spray Influenza Vaccine "FluMist" for Self- or Caregiver-Administration

"FDA approve first influenza vaccine for self- or caregiver-administration provides a new option for receiving a safe and effective seasonal influenza vaccine potentially with greater convenience, flexibility and accessibility for individuals and families,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Getting vaccinated each year is the best way to prevent influenza, which causes illness in a substantial proportion of the U.S. population every year and may result in serious complications, including hospitalization and death. This approval adds another option for vaccination against influenza disease and demonstrates the FDA’s commitment to advancing public health.”

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-nasal-spray-influenza-vaccine-self-or-caregiver-administration

Quote from Chief Pharmacist on October 5, 2024, 8:36 am

FDA Approves First Treatment Miplyffa (arimoclomol) for Niemann-Pick Disease, Type C

“NPC is a serious disease that leads to enormous adverse impacts on patients and families. Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients,” said Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM), in the FDA’s Center for Drug Evaluation and Research. “The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-niemann-pick-disease-type-c

Quote from Chief Pharmacist on October 7, 2024, 11:37 am

FDA Approves First Nasal Spray Neffy for Treatment of Anaphylaxis

"FDA approve the first epinephrine product for the treatment of anaphylaxis that is not administered by injection. Anaphylaxis is life-threatening and some people, particularly children, may delay or avoid treatment due to fear of injections,” said Kelly Stone, MD, PhD, Associate Director of the Division of Pulmonology, Allergy and Critical Care in the FDA’s Center for Drug Evaluation and Research. “The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis. As a result, neffy provides an important treatment option and addresses an unmet need.”

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-first-nasal-spray-treatment-anaphylaxis

Quote from Chief Pharmacist on October 7, 2024, 11:42 am

FDA Approves COBENFY with New Mechanism of Action for Treatment of Schizophrenia.

“Schizophrenia is a leading cause of disability worldwide. It is a severe, chronic mental illness that is often damaging to a person’s quality of life,” said Tiffany Farchione, M.D., director of the Division of Psychiatry, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “This drug takes the first new approach to schizophrenia treatment in decades. This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed.”

Source: https://www.fda.gov/news-events/press-announcements/fda-approves-drug-new-mechanism-action-treatment-schizophrenia