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Oct 15Thu
  1. biospace.com62

    FDA's latest rare disease framework seeks to prioritize consistency in flexibility

    The FDA launched its Rare Disease Framework Initiative to standardize how clinical trial requirements are applied across rare diseases while tailoring evidence expectations to what small patient populations can realistically generate. Only 5% of an estimated 10,000 rare conditions affecting 30 million Americans have an FDA-approved treatment. The framework builds on programs such as the Rare Disease Innovation Hub, CDER's ARC program and the RDEA pilot, and follows the November 2025 plausible mechanism pathway. The FDA will hold a meeting on Oct. 28 to gather feedback from patients, advocates and industry.

    Why it matters: FDA policy shift sets consistent rules for rare disease drug development, a core issue for sponsors of gene and cell therapies.

  2. ema.europa.eu90

    EMA recommends authorisation of Rasonque for metastatic pancreatic cancer

    EMA's CHMP has recommended EU marketing authorisation for Revolution Medicines' Rasonque (daraxonrasib), an oral RAS inhibitor for adults with metastatic pancreatic adenocarcinoma previously treated at least once. In a 500-patient phase 3 study, median overall survival was 12.4 months versus 6.7 with chemotherapy, and median progression-free survival 7.2 versus 3.6 months. The European Commission will now fast-track its decision, with pricing left to Member States.

    Why it matters: CHMP backing via a phased review under 150 days sets precedent for faster EU assessments of priority cancer medicines.

  3. medicalxpress.com53

    Expanded genetic testing and early transplant referrals anchor new myelofibrosis care recommendations

    The American Society of Hematology has issued 10 evidence-based recommendations for managing myelofibrosis, a rare blood cancer affecting up to six per 100,000 people, published in Blood Advances. The guidelines call for extended molecular testing at diagnosis, early transplant consultation for higher-risk patients responding to JAK inhibitors, appropriate JAK inhibitor use by risk level, anemia management strategies, and low-dose aspirin for patients with the JAK2 V617F mutation. Panel chair Brady Stein says the panel hopes the questions posed will set a future research agenda on treatment prediction, anemia and transplant timing.

    Why it matters: First ASH guidelines covering myelofibrosis care decisions from testing to transplant will shape clinical practice in a rare cancer.

Oct 14Wed
  1. fda.gov91

    FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer

    The FDA expanded approval of Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, to adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer, the first FDA-approved treatment for that disease. The decision relied on evidence from Anktiva's earlier approval for carcinoma in situ, where a trial showed a majority of adults achieved a complete response and many maintained it for at least 12 months. The FDA is finalizing draft guidance to let developers use CIS study evidence for papillary-only NMIBC approvals.

    Why it matters: FDA extends Anktiva to BCG-unresponsive papillary-only NMIBC and sets a precedent for cross-indication evidence use.

  2. fda.gov76

    FDA approves an expanded indication for nogapendekin alfa inbakicept-pmln for BCG-unresponsive non-muscle invasive bladder cancer

    On October 14, 2026, the FDA expanded approval of nogapendekin alfa inbakicept-pmln (Anktiva, Altor BioScience, an ImmunityBio subsidiary) with BCG to cover adults with BCG-unresponsive non-muscle invasive bladder cancer with high-grade papillary-only tumors, beyond the prior indication limited to carcinoma in situ. The agency extrapolated efficacy from Cohort A of QUILT-3.032, a single-arm trial of 77 patients, which showed a complete response rate of 62%. The recommended dose is 400 mcg intravesically with BCG weekly for 6 weeks induction, followed by maintenance for up to 37 months.

  3. fda.gov45

    Jiangmen Shuizirun Sanitary Articles Co., Ltd. - 722974 - 09/23/2026

    FDA issued a warning letter, reference 320-26-130, to Jiangmen Shuizirun Sanitary Articles Co., Ltd. of Guangdong, China, after a November 3 to 7, 2025 inspection of its drug manufacturing facility documented CGMP violations for finished pharmaceuticals. The firm, whose OTC products are labeled sterile but not sterilized and lack sterility testing at release, also failed to conduct identity testing on incoming components and to validate supplier certificates of analysis. The letter, sent September 23, 2026, asks for an action plan covering customer notifications, recalls or market withdrawals for sterile-labeled batches in U.S. distribution.

  4. fda.gov55

    Hanlim Pharm Co., Ltd. - 734782 - 10/02/2026

    FDA issued a warning letter, reference 320-27-03, to Hanlim Pharm Chairman Jungjin Kim on October 2, 2026, citing significant CGMP violations at the company's sterile drug manufacturing facility in Yonginsi, Gyeonggi-do, inspected from April 16 to April 24, 2026. The letter details poor aseptic practices, inadequate smoke studies and cleanroom design flaws risking product sterility, and calls the company's May 15, 2026 response inadequate. Hanlim must now submit a systematic plan covering aseptic practices, cleanroom behavior and independent assessments, or face seizure or injunction.

    Why it matters: FDA warning letter over CGMP violations at a sterile drug manufacturing site affects Hanlim Pharm's US supply prospects.

  5. fda.gov51

    Eurofins SF Analytical Laboratories, Inc. - 736056 - 10/06/2026

    FDA issued a warning letter dated October 6, 2026 to Eurofins SF Analytical Laboratories, a contract testing laboratory in Horsham, Pennsylvania, after a June 1-3, 2026 inspection documented CGMP violations under 21 CFR parts 210 and 211. FDA found analysts selectively omitted too-numerous-to-count results, recorded counts from cracked and desiccated plates, and lacked secondary verification of zero-CFU readings; the firm admitted inconsistent reporting practices dating back to February 2025. FDA deemed the firm's June 22, 2026 response inadequate and demanded an independent laboratory assessment, a remediation plan, and a CAPA plan, warning that failure to comply may bring seizure or injunction.

  6. fda.gov49

    Silgan Dispensing Systems - 734538 - 10/05/2026

    FDA issued a warning letter dated October 5, 2026, to Earl Lawson, president of Silgan Dispensing Systems, after a March 26 to April 2, 2026 inspection of the Silgan Unicep Packaging facility in Spokane, Washington, which contract manufactures non-sterile over-the-counter drug products. FDA cited CGMP violations including invalidated out-of-specification microbiological results obtained through averaging, an inadequate investigation linked to a customer's Class I recall over potential fungal contamination, and unapproved new drug claims found in product labeling reviewed in July 2026. FDA reviewed the firm's April 22, 2026 response and warned that failure to correct the violations may result in seizure or injunction.

  7. fda.gov49

    Lovelace Biomedical Research Institute - 725136 - 10/06/2026

    FDA issued a Warning Letter to Lovelace Biomedical Research Institute in Albuquerque, citing Good Laboratory Practice violations found during an April 7-18, 2025 inspection under the Bioresearch Monitoring Program. The quality assurance unit failed to review final study reports so that results reflected raw data, omitting abnormal clinical observations in 20 of 34 animals in one protocol and dosing incidents such as pulled catheters and infiltration at the infusion site. FDA found the May 9, 2025 written response and the revised reports dated May 30, 2025 inadequate.

  8. fda.gov50

    SCA Pharmaceuticals, LLC - 724202 - 09/14/2026

    FDA issued a warning letter to SCA Pharmaceuticals, LLC, a 503B outsourcing facility in Little Rock, Arkansas, on September 14, 2026, following an inspection from October 20 to 30, 2025 that found sterile drug products prepared under insanitary conditions. Cited deficiencies include inadequate ISO 5 certification, poor response to microbial contamination, and aseptic technique failures during production of lots such as Fentanyl Citrate 2 mcg/mL Bupivacaine HCl 0.125% and Methadone HCl 10 mg/mL. FDA reviewed the firm's responses of November 21 and December 31, 2025, and deemed the products adulterated under section 501(a)(2)(A) of the FDCA.

Oct 13Tue
  1. biocentury.com48

    Overton confirmation battling clock, abortion politics

    Heidi Overton's confirmation as FDA commissioner faces a tight timeline, Republican divisions over abortion, and Trump administration vaccine policies. Senator Hawley's concerns about an FDA study of mifepristone safety complicate the process, and Republicans hold a one-seat majority on the Senate HELP Committee, so Overton cannot lose any Republican votes if all Democrats oppose her.

  2. nature.com84

    RevMed’s pancreatic cancer drug first

    The FDA approved Revolution Medicines' Rasonque (daraxonrasib) in August for metastatic pancreatic adenocarcinoma, making it a first-in-class RAS inhibitor for the most common form of pancreatic cancer. Rasonque uses a molecular glue mechanism, binding cyclophilin A and RAS in its GTP-bound 'on' state, unlike KRAS G12C inhibitors such as Lumakras and Krazati. An early-stage trial in September showed promise in lung cancer, raising hopes for use in other hard-to-treat tumors.

    Why it matters: First FDA approval of a pan-RAS inhibitor for metastatic pancreatic adenocarcinoma, with early lung cancer activity.

  3. nature.com80

    FDA nod for rare neurology drug

    The FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen), the first disease-modifying drug for Alexander disease, a rare progressive and often fatal neurological condition affecting about one person per 1-3 million. The antisense oligonucleotide targets RNA encoding glial fibrillary acidic protein (GFAP), reducing its overproduction in astrocytes, where excess GFAP forms clumps that disrupt myelination. Patients suffer impaired motor function, seizures, intellectual disability and developmental delays.

    Why it matters: First disease-modifying therapy approved for a rare, previously untreatable neurological condition.

Oct 10Sat
  1. fda.gov87

    FDA Approves Drug to Treat Lesions Caused by Herpes Simplex Virus

    FDA has approved Hovilpri (pritelivir) tablets for immunocompromised adults with mucocutaneous herpes simplex virus lesions who did not respond to acyclovir, valacyclovir or famciclovir. In the Phase 3 PRIOH-1 Part C trial of 101 adults, all lesions healed within 28 days in 63% of pritelivir patients versus 34% on investigator-chosen treatments. The drug received priority review, and headache was the most common adverse reaction.

    Why it matters: First approval of a new mechanism option for immunocompromised HSV patients resistant or intolerant to existing antivirals.

  2. fda.gov40

    Novel Drug Approvals for 2026

    The FDA's novel drug approvals page for 2026 lists 46 approvals to date, from relacorilant for platinum-resistant ovarian cancer on 3/25 through pritelivir for refractory mucocutaneous herpes simplex lesions in immunocompromised adults on 10/9. The list spans oncology, rare disease, metabolic and imaging agents, including orforglipron for obesity, tulisokibart for ulcerative colitis, and two FOP drugs, zilurgisertib and garetosmab. The list is updated as further approvals are granted.

Oct 9Fri
  1. fda.gov49

    Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products: Questions and Answers

    The FDA has issued a final guidance for industry, "Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products: Questions and Answers," from its drug and biologics centers. It answers common questions on postapproval manufacturing changes for biosimilar and interchangeable biosimilar products licensed under the PHS Act. The guidance finalizes a draft of the same title issued on July 23, 2024, under docket FDA-2024-D-2581, and comments may be submitted at any time.

  2. biocentury.com40

    FDA’s CDER approved nine NMEs in September

    FDA's CDER approved nine new molecular entities in September 2026, its highest monthly total of the year, lifting the year-to-date count to 45, one short of the full-year 2025 figure. Six of the nine approvals were treatments for rare diseases, including first approved therapies for MCT8 deficiency and Alexander disease. Companies behind the approvals include Abbvie, AstraZeneca, Bristol Myers Squibb, Curium Pharma, Egetis Therapeutics, Eisai and Elevar Therapeutics.

  3. fda.gov93

    FDA approves atezolizumab in combination with chemotherapy for Stage III mismatch repair deficient colon cancer

    The FDA approved atezolizumab (Tecentriq, Genentech) in combination with a fluoropyrimidine and oxaliplatin on October 8, 2026, for adjuvant treatment of Stage III mismatch repair deficient colon cancer in patients two years of age and older, with the subcutaneous Tecentriq Hybreza approved for those 12 years and older weighing 40 kg or greater. In the ATOMIC trial of 711 adults and one pediatric patient, the hazard ratio for disease-free survival was 0.50 versus mFOLFOX6 alone, and median DFS was not reached in either arm. Reviews are ongoing at partner agencies under Project Orbis.

    Why it matters: FDA approval of atezolizumab plus chemotherapy as adjuvant therapy for Stage III dMMR colon cancer, with a disease-free survival hazard ratio of 0.50.

Oct 8Thu
  1. fda.gov80

    FDA approves tucatinib with trastuzumab and pertuzumab for the maintenance treatment of HER2-positive breast cancer

    The FDA approved tucatinib (Tukysa, Seagen, a Pfizer subsidiary) with trastuzumab and pertuzumab on October 7, 2026, for maintenance treatment of adults with unresectable locally advanced or metastatic HER2-positive breast cancer after induction therapy. In the 654-patient HER2CLIMB-05 trial, median progression-free survival was 24.9 months with tucatinib 300 mg twice daily versus 16.3 months with placebo (HR 0.64, p<0.0001). The label carries a boxed warning for hepatotoxicity; overall survival data were immature.

    Why it matters: FDA approval extends tucatinib to first-line maintenance, with PFS results from a randomized phase 3 trial.

  2. fda.gov55

    Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing

    The FDA's Center for Drug Evaluation and Research issued a draft guidance on studying pharmacokinetics, and where appropriate pharmacodynamics, in patients with impaired hepatic function, including for therapeutic biological products. The non-binding recommendations cover study design, data analysis and impact on dosing for sponsors and applicants. Comments are due by 12/01/2026 under docket FDA-2026-D-8693 before work begins on the final version.

    Why it matters: Draft FDA guidance sets expectations for hepatic impairment PK studies that shape sponsors' clinical development plans.

Oct 7Wed
  1. fda.gov40

    Rare Disease Drug Approvals

    FDA's CDER lists recent rare disease drug approvals on its Accelerating Rare Disease Cures Program page. In 2026 alone these include Camzyos for an inherited heart condition in children, Emcitate for MCT8 deficiency, Isembyld for muscle loss in spinal muscular atrophy, and Zanvastro for Alexander Disease, among others. The list spans 2021 to 2026 and links to the underlying FDA news releases.

Oct 6Tue
  1. biospace.com48

    FDA extends review of Novo’s hemophilia A drug, citing manufacturing issues

    The FDA has extended its review of Novo's denecimig, an investigational bispecific antibody and next-gen factor VIII mimetic for prophylactic treatment of hemophilia A, citing ongoing facility remediation activities at an unnamed manufacturing site. The agency flagged no efficacy or safety deficiencies, and Novo, which submitted the drug in September 2025 with a decision originally slated for the third quarter, says it is addressing the FDA's requests and still preparing for a 2027 launch in adults and children. The FDA has not shared a new review timeline, and European regulators are also evaluating the programme.

    Why it matters: FDA manufacturing delay affects a potential hemophilia A approval and ties into Novo's broader Catalent-related quality problems.

  2. fda.gov44

    Online Generic Medicine/www.onlinegenericmedicine.com - 737806 - 09/23/2026

    FDA's Center for Drug Evaluation and Research sent a warning letter dated September 23, 2026 to Online Generic Medicine (www.onlinegenericmedicine.com) for selling unapproved and misbranded injectable semaglutide, including a product marketed as "Semanat Injection (Semaglutide)". The letter cites violations of sections 301(a), 301(d), 301(k), 502(f)(1), 503(b)(1) and 505(a) of the FD&C Act, identified in website reviews on August 26 and September 2, 2026. FDA asks the site to stop offering unapproved and misbranded drugs to U.S. consumers and warns that failure to comply may bring seizure or injunction.

  3. fda.gov46

    TCI Co., Ltd BioCosme PABP BRANCH - 736888 - 09/02/2026

    FDA issued a warning letter to TCI Co., Ltd BioCosme PABP BRANCH, a contract manufacturer of over-the-counter drugs in Pingtung County, Taiwan, citing significant CGMP violations found during an inspection from March 9 to 13, 2026. The firm released OTC drug batches to the U.S. market without assay testing of the active ingredient and failed to perform identity testing on incoming APIs and raw materials or verify supplier certificates of analysis. FDA found the firm's April 2, 2026 response to its Form FDA 483 inadequate and required a comprehensive quality agreement and lists of test methods and specifications; failure to correct the violations may bring regulatory action without further notice.

    Why it matters: FDA warning letter documents CGMP failures at an OTC contract manufacturer supplying the U.S. market.

  4. fda.gov46

    Pine Pharmaceuticals, LLC - 728537 - 08/07/2026

    FDA issued a warning letter dated August 7, 2026 to Pine Pharmaceuticals, LLC, a section 503B outsourcing facility in Tonawanda, New York, after a November 4-14, 2025 inspection found insanitary conditions and CGMP violations that rendered sterile drug products adulterated. Cited failures include inadequate disinfection of materials entering the ISO 5 hood, deficient environmental monitoring of staging trays, and incomplete batch failure investigations. The letter follows the firm's December 8, 2025 response to a Form FDA 483, and FDA expects corrective action.

  5. fda.gov55

    Walmart, Inc. - 736557 - 10/01/2026

    FDA's Center for Drug Evaluation and Research issued a warning letter dated October 1, 2026 to Walmart President and CEO John Furner over three products sold on walmart.com: HClear for him, Herstat GST, and Terragen HG Formula. Purchased by FDA in July 2026 and shipped via Walmart Fulfillment Services, the products are marketed for genital herpes and genital sore symptoms and are unapproved new drugs under section 505(a) of the FD&C Act. FDA notes no OTC drug product is approved for treating any STI and warns that failure to correct the violations may bring seizure or injunction without further notice.

    Why it matters: FDA targets a major retailer for selling unapproved STI products, a direct compliance risk for online drug marketplaces.

  6. fda.gov41

    Aidance Scientific, Inc. - 735346 - 10/01/2026

    FDA's Center for Drug Evaluation and Research issued a warning letter dated October 1, 2026 to Aidance Scientific, Inc. president Perry Antelman, citing the company's "Terragen HG Formula" as an unapproved new drug sold via its website and Amazon storefront for herpes and genital sores. The agency found no approved application for the product and warned that OTC products marketed for STIs pose public health risks, noting homeopathic drugs face the same approval requirements. Failure to correct the violations may prompt seizure or injunction.

  7. fda.gov42

    The Platinum Healthcare LLC - 735147 - 10/01/2026

    FDA's Center for Drug Evaluation and Research issued a warning letter on October 1, 2026 to The Platinum Healthcare LLC and its CEO Jamey Hagan of Pulaski, Tennessee, over products sold on its website. The agency found the homeopathic products "Sycosis" and "Syphillinum," marketed for gonorrhea and syphilis miasms, to be unapproved new drugs violating sections 301(d) and 505(a) of the FD&C Act, noting no OTC product is approved for treating STIs. The company must send a written response to FDA within fifteen business days or face possible seizure and injunction.

  8. fda.gov45

    DesBio/Deseret Biologicals, Inc. - 735464 - 10/01/2026

    FDA's Center for Drug Evaluation and Research sent a warning letter dated October 1, 2026 (reference 735464) to DesBio/Deseret Biologicals CEO Jake Carter, citing the product "CHLA:10M," sold on desbio.com for relief of chlamydia-related symptoms, as an unapproved new drug under sections 301(d) and 505(a) of the FD&C Act. The letter follows a website review in July 2026 and notes that FDA has not approved any OTC drug product for treating or preventing any STI. The company must send a written response to FDAAdvisory@fda.hhs.gov within 15 business days or face possible seizure or injunction.

  9. fda.gov45

    Bioactive Services USA, LLC / Herstat Inc. - 734784 - 10/01/2026

    FDA's Center for Drug Evaluation and Research issued a warning letter on October 1, 2026 to Kevin Aspinall Nessling, CEO of Bioactive Services USA, LLC / Herstat Inc., over the product "Herstat GST", marketed for genital sore treatment on the websites drsosnowski.com and herstat.com. FDA found the product is an unapproved new drug under section 505(a) of the FD&C Act, based on a July 2026 review of the websites and the firm's Facebook and Instagram accounts. FDA notes it has not approved any OTC drug for treating or preventing STIs, and warned that failure to correct the violations may result in seizure or injunction.

  10. fda.gov56

    Amazon.com, Inc. - 736006 - 10/01/2026

    FDA's Center for Drug Evaluation and Research sent a warning letter dated October 1, 2026 (reference 736006) to Amazon CEO Andy Jassy, citing nine products sold on amazon.com and fulfilled via Fulfillment by Amazon, including FemiClear, HClear for him, HerpCare Ointment and Spray, and RoyceDerm Herpes Relief Cream, as unapproved new drugs for genital herpes under section 505(a) of the FD&C Act. FDA, which purchased the products in July 2026, notes no OTC drug is approved for treating any STI and warns that reliance may delay care and risk infertility, sepsis and death. Amazon must correct the violations or face regulatory or legal action including seizure and injunction.

    Why it matters: FDA warning letter puts Amazon's marketplace and Fulfillment by Amazon service directly in the regulatory line for unapproved STI drugs.

  11. fda.gov45

    OrganiCare, LLC / OrganiCare Nature’s Science, LLC - 734925 - 10/01/2026

    FDA's Center for Drug Evaluation and Research sent a warning letter dated October 1, 2026 to Caroline Goodner, CEO of OrganiCare, LLC / OrganiCare Nature's Science, LLC, after a July 2026 review of the femiclear.com website and the company's Facebook and Instagram accounts. The letter states that "HClear for him" and "FemiClear Genital Herpes Symptoms" are unapproved new drugs under section 505(a) of the FD&C Act, marketed without any FDA-approved OTC product for treating STIs. FDA warns that failure to correct the violations may result in seizure and injunction without further notice.

  12. medicalxpress.com51

    Hormonal therapy recommended as a first-line treatment for menopausal vasomotor symptoms

    The American College of Physicians issued a clinical guideline in Annals of Internal Medicine recommending estrogen plus progestogen for females with a uterus, or estrogen alone for those without, as first-line treatment of menopausal vasomotor symptoms (hot flashes and night sweats), which affect up to 80% of females. For those with contraindications or intolerance to estrogen, ACP suggests an SNRI (desvenlafaxine or venlafaxine) as second-line treatment, then an SSRI, gabapentin or an NKra (elinzanetant or fezolinetant) as third-line. It urges clinicians to ask patients about vasomotor symptoms and weigh benefits, harms, cost and access in shared decisions.

    Why it matters: New ACP guideline sets a ranked order for hormonal and nonhormonal treatment of menopausal vasomotor symptoms.

  13. fda.gov40

    Rare Disease News & Events

    The FDA's ARC Program reviewed its 2025 rare disease work, reporting more than 30 public speaking engagements, 57-plus workshops, consults or meetings supporting rare disease drug development, and 45,000 rare disease news subscribers served. CDER approved 46 novel drugs in 2025, with 32 approved first in the U.S. and 23 carrying orphan drug designation. In FY25 the Office of Orphan Products Development granted 450 of 657 original orphan-drug designation requests, and public workshops on rare disease data sharing and neonatal product development are scheduled or archived for 2026.

  14. fda.gov47

    Glenmark Pharmaceuticals Limited - 637314 - 11/22/2022

    The FDA issued a warning letter to Glenmark Pharmaceuticals on November 22, 2022, after inspecting its finished-drug manufacturing facility in Colvale, Goa, from May 12 to May 20, 2022, and finding significant CGMP violations. The letter cites inadequate investigation of out-of-specification results, including 14 rejected batches of desmopressin acetate tablets from 2018 to 2021 and a June 10, 2022 voluntary recall of batch 20220121, plus insufficient process validation for a gel product with five batches rejected for viscosity failures between 2020 and 2021. Glenmark must respond with independent assessments and remediation plans for its investigation and CAPA systems and its process validation program.

  15. fda.gov64

    M15 Resource Center

    The FDA has posted a resource center for ICH M15, a guidance on general principles for model-informed drug development (MIDD). The guidance describes MIDD principles, including planning, model evaluation and evidence documentation, sets a harmonized framework for assessing MIDD-derived evidence, and gives recommendations on MIDD-related regulatory interactions, reporting and submission. FDA recommends sponsors use it when preparing meeting requests, background packages and regulatory submissions.

    Why it matters: FDA now expects ICH M15 principles to shape MIDD evidence in submissions and meetings.

  16. fda.gov54

    Expedited Investigational New Drug Pilot Program - Application Instructions

    The FDA has issued application instructions for its Expedited IND Pilot Program. Sponsors and prospective Qualified Research Institutions each complete a form, then the sponsor submits both through the FDA ESG Gateway as one package. The application window stays open for 45 days with possible extension, and FDA scientific experts will review submissions and select 8 to 10 Sponsor-QRI pairs. Participating parties must form their business relationship outside FDA, which will only confirm that an official agreement exists.

    Why it matters: FDA sets out how sponsors and QRIs apply for the Expedited IND Pilot Program and how many pairs will be selected.

Oct 5Mon
  1. statnews.com43

    STAT+: Lilly’s Jaypirca approved as first-line CLL therapy

    The FDA has approved Eli Lilly's Jaypirca as a first-line therapy for chronic lymphocytic leukemia (CLL), according to STAT News. The approval moves Jaypirca, already known in CLL treatment discussions, into the earlier-line setting. Details of the decision are behind the STAT+ paywall, with further coverage available to subscribers.

    Why it matters: First-line CLL approval expands the eligible patient population for Lilly's Jaypirca.

  2. nature.com73

    Gene therapy for rare lysosomal storage disorder nabs FDA approval

    The FDA has approved Ultragenyx Pharmaceutical's gene therapy rebisufligene etisparvovec (Fayuvi) for paediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. It is the first FDA-approved treatment option for this rare neurodegenerative lysosomal storage disorder.

    Why it matters: First FDA-approved therapy for a rare neurodegenerative lysosomal storage disorder.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org