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New Request for Applications for FDA Rare Neurodegenerative Disease Grant Program Deadline to submit applications: October 22, 2024 The U.S. Food and Drug Administration (FDA) announced a new funding opportunity for the FDA Rare Neurodegenerative Disease Grant Program to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare neurodegenerative diseases for children and adults. Through the support of collaborative, efficient, and innovative clinical trials, FDA hopes to exert a broad and positive impact by increasing the number of approved medical products for rare neurodegenerative diseases. The FDA Rare Neurodegenerative Disease Grant Program was established by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. This program is administered by FDA’s Office of Orphan Products Development to promote medical product development for rare neurodegenerative diseases such as ALS.  #fda #rarediseases #program #neurodegenerative #acceleratedaccess #deadline https://lnkd.in/g_SQN-2s

Accelerating Rare disease Cures (ARC) Program by FDA Next issue of CDER’s Accelerating Rare disease Cures (ARC) Program quarterly newsletter as FDA celebrates the ARC Program's second anniversary! This is the platform to share updates about work and highlight upcoming event participation and engagement opportunities. Since the program launch in May of 2022, the ARC Program has built on CDER’s existing capabilities by fostering collaboration within the rare disease stakeholder community. https://lnkd.in/gdPBD8B7

FDA approves lazertinib with amivantamab-vmjw for non-small lung cancer  Food and Drug Administration approved lazertinib (Lazcluze, Janssen Biotech, Inc.) in combination with amivantamab-vmjw (Rybrevant, Janssen Biotech, Inc.) for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test. https://lnkd.in/gcYP7gCS

Guidance for Industry: PSG Meetings Between FDA and ANDA Applicants FDA is committed to ensuring current and prospective ANDA applicants have the information they need to successfully submit high-quality ANDAs. On August 19, 2024, the agency published the guidance for industry, “Product-Specific Guidance Meetings Between FDA and ANDA Applicants Under GDUFA.” This guidance finalizes the draft version published in February 2023.  The guidance provides: Recommendations on product-specific guidance (PSG) meetings between FDA and prospective applicants or applicants that have submitted abbreviated new drug applications (ANDAs) Information on requesting and conducting PSG meetings with FDA (i.e., pre-submission PSG teleconferences, post-submission PSG teleconferences, pre-submission PSG meetings, and post-submission PSG meetings), which are outlined in the GDUFA III Commitment Letter Procedures for well-managed PSG meetings to help ensure meetings are scheduled and conducted in accordance with the timeframes set forth in the GDUFA III Commitment Letter Changes from the draft version of the guidance, issued in February 2023, to the final guidance published today include: Updates to clarify when applicants can submit PSG teleconference and PSG meeting requests Topics that applicants can discuss during PSG teleconferences and PSG meetings When applicants should utilize other pathways to seek FDA’s feedback Editorial changes to improve clarity FDA recommends that applicants consult published PSGs when considering an appropriate bioequivalence study and/or other studies for a proposed drug product. PSG Support for ANDA Applicants PSGs provide recommendations for developing generic drug products and describe FDA’s current thinking on the evidence needed to demonstrate that an ANDA is therapeutically equivalent to a specific reference listed drug product. PSG teleconferences provide a forum for applicants to receive FDA’s feedback on the potential impact of a new or revised PSG on the applicant’s ANDA development program. #fda #guidance #communication #drugdevelopment #meeting #genericdrugs Pre-submission PSG meetings and post-submission PSG meetings provide a forum in which applicants can discuss the scientific rationale for an approach other than the approach recommended in the PSG to ensure that the approach complies with the relevant statutes and regulations. https://lnkd.in/dn4u8-fw

FDA approves neoadjuvant/adjuvant durvalumab for resectable non-small cell lung cancer On August 15, 2024, the Food and Drug Administration approved durvalumab (Imfinzi, AstraZeneca) with platinum-containing chemotherapy as neoadjuvant treatment, followed by single-agent durvalumab as adjuvant treatment after surgery for adults with resectable (tumors ≥ 4 cm and/or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. #fda #approval #oncology #immunotherapy #checkpointinhibitor #ici https://lnkd.in/gFYNNzUD

9 Things to Know About CDER’s Efforts on Rare Diseases The Orphan Drug Act defines a rare disease as any disease or condition that affects less than 200,000 people in the U.S. There are approximately 25 to 30 million Americans living with a rare disease (about 1 in 10 people). Many rare conditions are life-threatening, and most do not have treatments. Accelerating the development of safe and effective drugs is CDER’s core mission, and CDER understands there are unique challenges in demonstrating the safety and effectiveness of drugs that treat rare diseases. #fda #raredisease #facts https://lnkd.in/gxwtqdVB

FDA approves axatilimab-csfr for chronic graft-versus-host disease Food and Drug Administration approved axatilimab-csfr (Niktimvo, Incyte Corporation), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. #fda #approval #immunotherapy https://lnkd.in/gQrubK9x

FDA approves treatment for primary biliary cholangitis FDA has approved Livdelzi (seladelpar) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.   PBC is a rare autoimmune disease in which the small bile ducts in the liver become injured and inflamed and without treatment are eventually destroyed. Most patients are females who are diagnosed with PBC between 40 and 60 years of age; without treatment they experience a slow progression of liver disease to cirrhosis, liver failure, and potential need for liver transplantation or death.  The recommended dosage is 10 mg orally once daily. The most common adverse reactions reported with use of Livdelzi are headache, abdominal pain, nausea, abdominal distension, and dizziness. Use of Livdelzi is not recommended in patients who have or who develop decompensated cirrhosis (e.g., ascites, variceal bleeding, hepatic encephalopathy). Providers should measure patients’ baseline liver tests before prescribing Livdelzi and monitor these tests during treatment. Providers should also be mindful of potential side effects of bone fractures. See full prescribing information for additional information on risks associated with Livdelzi.  Livdelzi received accelerated approval for this indication, and the manufacturer is required to conduct post-marketing studies to describe and verify clinical benefit. Livdelzi also received orphan drug designation and breakthrough therapy designation for this indication.    #fda #approval #orphandrug #biliarycholangitis https://lnkd.in/gTyGDUrH

FDA Approves New Drug for Hypoparathyroidism, a Rare Disorder The U.S. Food and Drug Administration has approved Yorvipath (palopegteriparatide) injection for subcutaneous use in adults with hypoparathyroidism. Yorvipath was not studied in adults with acute post-surgical hypoparathyroidism. Hypoparathyroidism is a rare disease most often caused by damage to the parathyroid glands from surgery or autoimmune disease. Patients with hypoparathyroidism have low levels of parathyroid hormone (PTH), which leads to hypocalcemia (blood calcium levels that are too low). Yorvipath’s labeling includes warnings for a potential risk of risk of unintended changes in serum calcium levels related to number of daily injections and total delivered dose, serious hypocalcemia and hypercalcemia (blood calcium levels that are too high), osteosarcoma (a rare bone cancer) based on findings in rats, orthostatic hypotension (dizziness when standing), and a risk of a drug interaction with digoxin (a medicine for certain heart conditions) #fda #drug #approval #raredisease https://lnkd.in/gXMHM4mv

Nasal spray of epinephrine was approved to treat anaphylaxis