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FDA Pipeline, October-December 2023

September 2023 Vol 16, Payers' Guide to FDA Updates - FDA Approvals
Table

References

  1. Standard FDA calendar. FDA Tracker. https://www.fdatracker.com/fda-calendar/. Accessed August 21, 2023.
  2. Alnylam Announces U.S. Food and Drug Administration (FDA) Acceptance of Supplemental New Drug Application for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis. Business Wire. Published February 21, 2023. https://www.businesswire.com/news/home/20230221005335/en/Alnylam-Announces-U.S.-Food-and-Drug-Administration-FDA-Acceptance-of-Supplemental-New-Drug-Application-for-ONPATTRO%C2%AE-patisiran-for-the-Treatment-of-the-Cardiomyopathy-of-ATTR-Amyloidosis. Accessed August 21, 2023.
  3. US Food and Drug Administration. FDA Approves First-of-Its Kind Targeted RNA-Based Therapy to Treat a Rare Disease. Published August 10, 2018. https://www.fda.gov/news-events/press-announcements/fda-approves-first-its-kind-targeted-rna-based-therapy-treat-rare-disease. Accessed August 21, 2023.
  4. ONPATTRO (Patisiran) Lipid Complex Injection, for Intravenous Use [Prescribing Information]. Alnylam Pharmaceuticals; 2018. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210922s000lbl.pdf. Accessed August 21, 2023.
  5. U.S. Food and Drug Administration Accepts Bristol Myers Squibb’s Supplemental Biologics License Application and European Medicines Agency Validates Application for Opdivo (nivolumab)….Business Wire. Published February 28, 2023. https://www.businesswire.com/news/home/20230227005793/en/U.S.-Food-and-Drug-Administration-Accepts-Bristol-Myers-Squibb%E2%80%99s-Supplemental-Biologics-License-Application-and-European-Medicines-Agency-Validates-Application-for-Opdivo-nivolumab-%E2%80%A6. Accessed August 21, 2023.
  6. OPDIVO (Nivolumab) Injection, for Intravenous Use [Prescribing Information]. Bristol-Myers Squibb Company; 2019. https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/125554s070lbl.pdf. Accessed August 21, 2023.
  7. Merck Announces Phase 3 KEYNOTE-671 Trial Met Primary Endpoint of Event-Free Survival (EFS) in Patients With Resectable Stage II, IIIA or IIIB Non-Small Cell Lung Cancer. Published March 1, 2023. https://www.businesswire.com/news/home/20230301005450/en/Merck-Announces-Phase-3-KEYNOTE-671-Trial-Met-Primary-Endpoint-of-Event-Free-Survival-EFS-in-Patients-With-Resectable-Stage-II-IIIA-or-IIIB-Non-Small-Cell-Lung-Cancer. Accessed August 21, 2023.
  8. KEYTRUDA® (Pembrolizumab) Injection, for Intravenous Use [Prescribing Information]. Merck & Co; 2018. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/125514s037lbl.pdf. Accessed August 21, 2023.
  9. VOXZOGO (Vosoritide) for Injection, for Subcutaneous Use [Prescribing Information]. BioMarin Pharmaceutical; 2021. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf. Accessed August 21, 2023.
  10. BioMarin Pharmaceutical Inc. FDA Accepts BioMarin’s Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5. Cision PR Newswire. https://www.prnewswire.com/news-releases/fda-accepts-biomarins-supplemental-new-drug-application-to-expand-use-of-voxzogo-vosoritide-for-injection-to-treat-children-with-achondroplasia-under-the-age-of-5-301765021.html. Accessed August 21, 2023.
  11. Regeneron Pharmaceuticals Inc. Dupixent® (dupilumab) Application for Treatment of Chronic Spontaneous Urticaria (CSU) in Adults and Adolescents Aged 12 Years and Older Accepted for FDA Review. GlobeNewswire News Room. Published March 7, 2023. https://www.globenewswire.com/news-release/2023/03/07/2621674/0/en/Dupixent-dupilumab-Application-for-Treatment-of-Chronic-Spontaneous-Urticaria-CSU-in-Adults-and-Adolescents-Aged-12-Years-and-Older-Accepted-for-FDA-Review.html. Accessed August 21, 2023.
  12. DUPIXENT® (Dupilumab) Injection, for Subcutaneous Use [Prescribing Information]. Regeneron Pharmaceuticals; 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761055s046lbl.pdf. Accessed August 21, 2023.
  13. Dupixent® (dupilumab) Application for Treatment of chronic Spontaneous Urticaria (CSU) in Adults and Adolescents accepted for FDA Review. US Securities and Exchange Commission. Published March 7, 2023. https://www.sec.gov/Archives/edgar/data/1121404/000119312523069910/d451411dex991.htm. Accessed August 21, 2023.
  14. Heron Therapeutics Announces Financial Results for the Three and Twelve Months Ended December 31, 2022 and Highlights Recent Corporate Updates. PR Newswire. Published March 23, 2023. https://www.prnewswire.com/news-releases/heron-therapeutics-announces-financial-results-for-the-three-and-twelve-months-ended-december-31-2022-and-highlights-recent-corporate-updates-301780323.html#:~:text=Heron's%20net%20loss%20for%20the,the%20same%20periods%20in%202021. Accessed August 21, 2023.
  15. Heron Therapeutics. ZYNRELEF (Heron Therapeutics, Inc.) instructions for use. MedLibrary.org. Published December 22, 2022. https://medlibrary.org/lib/rx/meds/zynrelef/page/11/. Accessed August 21, 2023.
  16. Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. JAMA Neurol. 2022;79(10):1005-1014.
  17. Santhera Pharmaceuticals Holding AG. Santhera Grants Exclusive North America License for Vamorolone to Catalyst Pharmaceuticals in Deal Valued at up to USD 231 Million Plus Royalties. GlobeNewswire News Room. Published June 20, 2023. https://www.globenewswire.com/news-release/2023/06/20/2690702/0/en/Santhera-Grants-Exclusive-North-America-License-for-Vamorolone-to-Catalyst-Pharmaceuticals-in-Deal-Valued-at-up-to-USD-231-Million-Plus-Royalties.html. Accessed August 21, 2023.
  18. Pacira Pharmaceuticals Inc. Pacira Announces FDA Acceptance of sNDA for Exparel Nerve Blocks to Produce Regional Analgesia in Lower Extremity Procedures. GlobeNewswire News Room. Published March 29, 2023. https://www.globenewswire.com/news-release/2023/03/29/2636622/0/en/Pacira-Announces-FDA-Acceptance-of-sNDA-for-Exparel-Nerve-Blocks-to-Produce-Regional-Analgesia-in-Lower-Extremity-Procedures.html. Accessed August 21, 2023.
  19. EXPAREL (Bupivacaine Liposome Injectable Suspension) [Prescribing Information]. Pacira Pharmaceuticals; 2018. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/022496s9lbl.pdf. Accessed August 21, 2023.
  20. Defencath (taurolidine/citrate/heparin) dosing, indications, interactions, adverse effects, and more. Medscape. https://reference.medscape.com/drug/defencath-taurolidine-citrate-heparin-4000151. Accessed August 21, 2023.
  21. CorMedix Inc. Announces FDA Acceptance of Resubmission of New Drug Application for DefenCath. US Securities and Exchange Commission. Published June 21, 2023. https://www.sec.gov/Archives/edgar/data/1410098/000121390023050240/ea180659ex99-1_cormedix.htm. Accessed August 21, 2023.
  22. VOQUEZNA TRIPLE PAK (Vonoprazan Tablets; Amoxicillin Capsules; Clarithromycin Tablets), Co-Packaged for Oral Use [Prescribing Information]. Phathom Pharmaceuticals; 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215152s000,215153s000lbl.pdf. Accessed August 21, 2023.
  23. Form 8-K (Phathom Pharmaceuticals, Inc.). US Securities and Exchange Commission. Published June 12, 2023. https://www.sec.gov/Archives/edgar/data/1783183/000119312523164930/d486645d8k.htm. Accessed August 21, 2023.
  24. Aldeyra Therapeutics Announces FDA Acceptance of New Drug Application for Reproxalap for the Treatment of Dry Eye Disease. US Securities and Exchange Commission. Published February 7, 2023. https://www.sec.gov/Archives/edgar/data/1341235/000115752323000181/a53304873_ex991.htm. Accessed August 21, 2023.
  25. Iovance Biotherapeutics Inc. Iovance Biotherapeutics Announces U.S. Food and Drug Administration Acceptance of the Biologics License Application of Lifileucel for the Treatment of Advanced Melanoma. GlobeNewswire News Room. Published May 26, 2023. https://www.globenewswire.com/news-release/2023/05/26/2677400/0/en/Iovance-Biotherapeutics-Announces-U-S-Food-and-Drug-Administration-Acceptance-of-the-Biologics-License-Application-of-Lifileucel-for-the-Treatment-of-Advanced-Melanoma.html. Accessed August 21, 2023.
  26. Sarnaik AA, Hamid O, Khushalani NI, et al. Lifileucel, a tumor-infiltrating lymphocyte therapy, in metastatic melanoma. J Clin Oncol. 2021;39(24):2656-2666.
  27. U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Repotrectinib for the Treatment of Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer. Business Wire. Published May 30, 2023. https://www.businesswire.com/news/home/20230528005015/en/U.S.-Food-and-Drug-Administration-Accepts-for-Priority-Review-Bristol-Myers-Squibb%E2%80%99s-Application-for-Repotrectinib-for-the-Treatment-of-Patients-with-Locally-Advanced-or-Metastatic-ROS1-Positive-Non-Small-Cell-Lung-Cancer. Accessed August 21, 2023.
  28. Yun MR, Kim DH, Kim SY, et al. Repotrectinib exhibits potent anti-tumor activity in treatment-naive and solvent-front-mutant ROS1-rearranged non-small cell lung cancer. Clin Cancer Res Off J Am Assoc Cancer Res. 2020;26(13):3287-3295.
  29. Turning Point Therapeutics, Inc. A Phase 1/2, Open-Label, Multi-Center, First-in-Human Study of the Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of TPX-0005 in Patients With Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements (TRIDENT-1). clinicaltrials.gov; 2023. https://clinicaltrials.gov/study/NCT03093116. Accessed August 20, 2023.
  30. SpringWorks Therapeutics Announces PDUFA Date Extension for Nirogacestat NDA. US Securities and Exchange Commission. Published June 5, 2023. https://www.sec.gov/Archives/edgar/data/1773427/000110465923067823/tm2317772d1_ex99-1.htm. Accessed August 21, 2023.
  31. HUTCHMED (China) Limited. Takeda and HUTCHMED Announce New Drug Application (NDA) for Fruquintinib for Treatment of Previously Treated Metastatic Colorectal Cancer Granted Priority Review. GlobeNewswire News Room. Published May 25, 2023. https://www.globenewswire.com/news-release/2023/05/25/2676813/0/en/Takeda-and-HUTCHMED-Announce-New-Drug-Application-NDA-for-Fruquintinib-for-Treatment-of-Previously-Treated-Metastatic-Colorectal-Cancer-Granted-Priority-Review.html. Accessed August 21, 2023.
  32. Form 8-K (Brainstorm Cell Therapeutics, Inc.). US Securities and Exchange Commission. Published June 6, 2023. https://www.sec.gov/Archives/edgar/data/1137883/000110465923068364/tm2317893d1_8k.htm. Accessed August 21, 2023.
  33. Lobo A. FDA final approval decision on NurOwn for ALS due by year’s end. ALS News Today. Published June 8, 2023. https://alsnewstoday.com/news/fda-final-approval-decision-brainstorms-nurown-als-due-december/. Accessed August 21, 2023.
  34. Maia M. FDA grants exa-cel priority review, with decision due by year’s end: Approval of CRISPR gene-editing therapy would be first for SCD. Sickle Cell Disease News. Published June 13, 2023. https://sicklecellanemianews.com/news/fda-priority-review-exa-cel-crispr-gene-editing-therapy-scd/. Accessed August 21, 2023.
  35. Form 8-K (CRISPR Therapeutics AG). US Securities and Exchange Commission. Published June 8, 2023. https://www.sec.gov/Archives/edgar/data/1674416/000095017023027129/crsp-20230608.htm. Accessed August 21, 2023.
  36. FDA Accepts Biologics License Applications for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. Published June 8, 2023. https://www.businesswire.com/news/home/20230608005819/en/FDA-Accepts-Biologics-License-Applications-for-exagamglogene-autotemcel-exa-cel-for-Severe-Sickle-Cell-Disease-and-Transfusion-Dependent-Beta-Thalassemia. Accessed August 21, 2023.
  37. LIVMARLITM (Maralixibat) Oral Solution. Mirum Pharmaceuticals; 2021. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/214662s001lbl.pdf. Accessed August 21, 2023.
  38. Form 10-Q (Mirum Pharmaceuticals, Inc.). US Securities and Exchange Commission. Published May 4, 2023. https://www.sec.gov/Archives/edgar/data/1759425/000095017023017879/mirm-20230331.htm. Accessed August 21, 2023.
  39. Arcutis Biotherapeutics Inc. Arcutis Announces FDA Acceptance of New Drug Application for Roflumilast Foam 0.3% for the Treatment of Seborrheic Dermatitis in Individuals Aged 9 Years and Older. GlobeNewswire News Room. Published April 18, 2023. https://www.globenewswire.com/news-release/2023/04/18/2648950/0/en/Arcutis-Announces-FDA-Acceptance-of-New-Drug-Application-for-Roflumilast-Foam-0-3-for-the-Treatment-of-Seborrheic-Dermatitis-in-Individuals-Aged-9-Years-and-Older.html. Accessed August 21, 2023.
  40. ABECMA® (Idecabtagene Vicleucel), Suspension for Intravenous Infusion [Prescribing Information]. Celgene Corporation; 2021. https://www.fda.gov/media/147055/download?attachment. Accessed August 21, 2023.
  41. Regulatory Applications Accepted Across Three Regions Globally for Abecma for Earlier Use in Adults with Triple-Class Exposed Relapsed and/or Refractory Multiple Myeloma. Business Wire. Published April 17, 2023. https://www.businesswire.com/news/home/20230413005797/en/Regulatory-Applications-Accepted-Across-Three-Regions-Globally-for-Abecma-for-Earlier-Use-in-Adults-with-Triple-Class-Exposed-Relapsed-andor-Refractory-Multiple-Myeloma. Accessed August 21, 2023.
  42. 2seventy bio Reports First Quarter Financial Results and Recent Operational Progress. Business Wire. Published May 3, 2023. https://www.businesswire.com/news/home/20230503005950/en/2seventy-bio-Reports-First-Quarter-Financial-Results-and-Recent-Operational-Progress. Accessed August 21, 2023.
  43. FDA Accepts Application for Merck’s KEYTRUDA® (pembrolizumab) Plus Chemotherapy as First-Line Treatment for Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma. Business Wire. Published April 13, 2023. https://www.businesswire.com/news/home/20230413005219/en/FDA-Accepts-Application-for-Merck%E2%80%99s-KEYTRUDA%C2%AE-pembrolizumab-Plus-Chemotherapy-as-First-Line-Treatment-for-Locally-Advanced-Unresectable-or-Metastatic-Gastric-or-Gastroesophageal-Junction-Adenocarcinoma. Accessed August 21, 2023.
  44. OptiNose Inc. Optinose Announces FDA Acceptance of Supplemental New Drug Application for XHANCE. GlobeNewswire News Room. Published May 4, 2023. https://www.globenewswire.com/news-release/2023/05/04/2661317/0/en/Optinose-Announces-FDA-Acceptance-of-Supplemental-New-Drug-Application-for-XHANCE.html. Accessed August 21, 2023.
  45. bluebird bio Announces FDA Priority Review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events. Business Wire. Published June 21, 2023. https://www.businesswire.com/news/home/20230621695509/en/bluebird-bio-Announces-FDA-Priority-Review-of-the-Biologics-License-Application-for-lovotibeglogene-autotemcel-lovo-cel-for-Patients-with-Sickle-Cell-Disease-SCD-12-years-and-Older-with-a-History-of-Vaso-Occlusive-Events. Accessed August 21, 2023.
  46. Glaukos Announces FDA Acceptance of NDA Submission for iDose TR. Business Wire. Published May 5, 2023. https://www.businesswire.com/news/home/20230505005442/en/Glaukos-Announces-FDA-Acceptance-of-NDA-Submission-for-iDose-TR. Accessed August 21, 2023.
  47. Ionis Pharmaceuticals Inc. Ionis Announces FDA Acceptance of New Drug Application for Eplontersen for the Treatment of Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN). Cision PR Newswire. Published March 7, 2023. https://www.prnewswire.com/news-releases/ionis-announces-fda-acceptance-of-new-drug-application-for-eplontersen-for-the-treatment-of-hereditary-transthyretin-mediated-amyloid-polyneuropathy-attrv-pn-301764027.html. Accessed August 21, 2023.
  48. Ionis Presents Positive Results From Phase 3 NEURO-TTRansform Study at International Symposium on Amyloidosis. Ionis Pharmaceuticals. Published September 7, 2022. https://ir.ionispharma.com/news-releases/news-release-details/ionis-presents-positive-results-phase-3-neuro-ttransform-study. Accessed August 21, 2023.
  49. Amgen. Amgen Reports Second Quarter Financial Results. Cision PR Newswire. Published August 3, 2023. https://www.prnewswire.com/news-releases/amgen-reports-second-quarter-financial-results-301893066.html. Accessed August 21, 2023.
  50. LUMAKRASTM (Sotorasib) Tablets, for Oral Use [Prescribing Information]. Amgen; 2021. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214665s000lbl.pdf. Accessed August 21, 2023.
  51. Merck Announces Second-Quarter 2023 Financial Results. US Securities and Exchange Commission. https://www.sec.gov/Archives/edgar/data/310158/000110465923086030/tm2322190d1_ex99-1.htm. Accessed August 21, 2023.
  52. Markham A. Gefapixant: first approval. Drugs. 2022;82(6):691-695.
  53. Smith JA, Kitt MM, Morice AH, et al. Gefapixant, a P2X3 receptor antagonist, for the treatment of refractory or unexplained chronic cough: a randomised, double-blind, controlled, parallel-group, phase 2b trial. Lancet Respir Med. 2020;8(8):775-785.
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Last modified: October 24, 2023