Regulatory Guidance Monthly Review - September 2023

Each month, Veristat's regulatory team tracks and shares a selection of new Regulatory Guidance updates from the US Food & Drug Administration (FDA)  that are pertinent to the governance of clinical trials and drug, biologic, or medical device development and approval. Below are the relevant links to updates provided for both COVID-19 related and non-COVID-19 related programs in September 2023.

FDA Final Guidance

Nontuberculous Mycobacterial Pulmonary Disease Caused by Mycobacterium avium Complex: Developing Drugs for Treatment (published 05-Sep-2023)
Download FDA Guidance

Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products (published 11-Sep-2023)
Download FDA Guidance

Annual Status Report Information and Other Submissions for Postmarketing Requirements and Commitments: Using Forms FDA 3988 and FDA 3989 Guidance for Industry (published 15-Sep-2023)
Download FDA Guidance

Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies (published 21-Sep-2023)
Download FDA Guidance

FDA Draft Guidance:

Endogenous Cushing’s Syndrome: Developing Drugs for Treatment (published 08-Sep-2023)
Draft FDA Guidance

Clinical Pharmacology Considerations for Peptide Drug Products (published 11-Sep-2023)
Draft FDA Guidance

Biosimilarity and Interchangeability: Additional Draft Q&As on Biosimilar Development and the BPCI Act (Revision 1) (published 18-Sep-2023)
Draft FDA Guidance

Labeling for Biosimilar and Interchangeable Biosimilar Products (published 18-Sep-2023)
Draft FDA Guidance

Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence (published 19-Sep-2023)
Draft FDA Guidance

Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products (published 22-Sep-2023)
Draft FDA Guidance

Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications (published 22-Sep-2023)
Draft FDA Guidance

Graft-versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment (published 28-Sep-2023)
Draft FDA Guidance

Monthly FDA Approvals

Approval Date: 09/07/23
Drug Name: XALKORINDA #217581
Active Ingredients: CRIZOTINIB
Submission Classification*:
Review Priority**: Standard
Company: PF PRISM CV

Approval Date: 09/08/23
Drug Name: APHEXDANDA #217159
Active Ingredients: MOTIXAFORTIDE
Submission Classification*: Type 1 - New Molecular Entity
Review Priority**: Standard
Company: BIOLINERX, LTD.

Approval Date: 09/12/2023
Drug Name: CYCLOPHOSPHAMIDENDA #217150
Active Ingredients: CYCLOPHOSPHAMIDE
Submission Classification*: Type 5 - New Formulation or New Manufacturer
Review Priority**: Standard
Company: SANDOZ INC

Approval Date: 09/14/2023
Drug Name: DOLUTEGRAVIR; TENOFOVIR DISOPROXIL FUMARATE; LAMIVUDINENDA #217016
Active Ingredients: DOLUTEGRAVIR; TENOFOVIR DISOPROXIL FUMARATE; LAMIVUDINE
Submission Classification*: New Combination and New Formulation or New Manufacturer
Review Priority**: Standard
Company: STRIDES PHARMA

Approval Date: 09/15/2023
Drug Name: OJJAARANDA #216873
Active Ingredients: MOMELOTINIB DIHYDROCHLORIDE
Submission Classification*: Type 1 - New Molecular Entity
Review Priority**: Standard
Company: GLAXOSMITHKLINE LLC

Approval Date: 09/22/2023
Drug Name: EXXUANDA #021164
Active Ingredients: GEPIRONE
Submission Classification*: Type 1 - New Molecular Entity
Review Priority**: Standard
Company: FABRE-KRAMER PHARMACEUTICALS, INC.

Approval Date: 09/22/2023
Drug Name: LIKMEZNDA #216755
Active Ingredients: METRONIDAZOLE
Submission Classification*: Type 5 - New Formulation or New Manufacturer
Review Priority**: Standard
Company: SAPTALIS PHARMACEUTICALS, LLC

Approval Date: 09/25/2023
Drug Name: RYZUMVINDA #217064
Active Ingredients: PHENTOLAMINE
Submission Classification*: Type 3 - New Dosage Form
Review Priority**: Standard
Company: OCUPHIRE PHARMA INC

Approval Date: 09/26/2023
Drug Name: BOSULIFNDA #217729
Active Ingredients: BOSUTINIB
Submission Classification*: Type 3 - New Dosage Form
Review Priority**: Priority
Company: PF PRISM CV

Approval Date: 09/27/2023
Drug Name: ENTYVIOBLA #761133
Active Ingredients: VEDOLIZUMAB
Submission Classification*:
Review Priority**:
Company: TAKEDA PHARMS USA

Approval Date: 09/28/2023
Drug Name: OPFOLDANDA #215211
Active Ingredients: MIGLUSTAT
Submission Classification*: Type 5 - New Formulation or New Manufacturer
Review Priority**: Standard
Company: AMICUS THERAP US

Approval Date: 09/29/2023
Drug Name: POMBILITIBLA #761204
Active Ingredients: CIPAGLUCOSIDASE ALFA-ATGA
Submission Classification*:
Review Priority**:
Company: AMICUS THERAP US

Approval Date: 09/29/2023
Drug Name: TECHNEGASNDA #022335
Active Ingredients: TECHNETIUM TC 99M CARBON
Submission Classification*: Type 3 - New Dosage Form
Review Priority**: Standard
Company: CYCLOMEDICA AUSTRALIA PTY LTD

Approval Date: 09/29/2023
Drug Name: RIVFLOZANDA #215842
Active Ingredients: NEDOSIRAN
Submission Classification*: Type 1 - New Molecular Entity
Review Priority**: Standard
Company: NOVO NORDISK INC

Approval Date: 09/29/2023
Drug Name: MICAFUNGINNDA #216142
Active Ingredients: MICAFUNGIN SODIUM
Submission Classification*: Type 5 - New Formulation or New Manufacturer
Review Priority**: Standard
Company: BAXTER HLTHCARE CORP

Approval Date: 09/29/2023
Drug Name: VASOPRESSIN IN SODIUM CHLORIDE INJECTIONNDA #217569
Active Ingredients: VASOPRESSIN IN SODIUM CHLORIDE INJECTION
Submission Classification*: Type 5 - New Formulation or New Manufacturer
Review Priority**: Standard
Company: BAXTER HLTHCARE CORP

Approval Date: 09/29/2023
Drug Name: TOFIDENCEBLA #761354
Active Ingredients: TOCILIZUMAB-BAVI
Submission Classification*:
Review Priority**:
Company: BIOGEN MA

Upcoming Advisory Board Meetings

10/04/2023- Meeting of the Oncologic Drugs Advisory Committee- 09:30 AM EDT to 03:00 PM EDT

10/05/2023- Vaccines and Related Biological Products Advisory Committee Meeting- 08:30 AM EDT- 01:00 PM EDT

10/31/2023- Cellular, Tissue, and Gene Therapies Advisory Committee Meeting- 09:00 AM EDT- 05:00 AM EDT

FDA publishes additional guidances on delay of enforcing DSCSA electronic tracking requirements (published 01-Sep-2023).
Read Article on RAPS.org

FDA finalizes guidance on standards for tracing products through supply chain (published 05-Sep-2023)
Read Article on RAPS.org

FDA finalizes guidance on use of human factors studies in combination product development (published 08-Sep-2023)
Read Article on RAPS.org

Perspective: FDA’s accelerated approval guidance doesn’t go far enough (published 11-Sep-2023)
Read Article on RAPS.org

FDA wants participants for CMC readiness pilot for expedited drugs (published 11-Sep-2023)
Read Article on RAPS.org

FDA revises guidance on IRB review of expanded access submissions to reflect end of public health emergency (published 12-Sep-2023)
Read Article on RAPS.org

FDA offers advice on developing drugs for endogenous Cushing’s syndrome (published 13-Sep-2023)
Read Article on RAPS.org

FDA issues draft guidance of clinical pharmacology, labeling considerations for peptide drug products (published 14-Sep-2023)
Read Article on RAPS.org

Biosimilar labeling guidance suggests cutting interchangeability details from labels (published 18-Sep-2023)
Read Article on RAPS.org

New OTP head discusses policy priorities for cell and gene therapies (published 19-Sep-2023)
Read Article on RAPS.org

FDA expands types of acceptable confirmatory evidence in new guidance (published 19-Sep-2023)
Read Article on RAPS.org

FDA guidance on conducting trials during emergencies shifts away from COVID-19 (published 21-Sep-2023)
Read Article on RAPS.org

FDA guidance on formal meetings adds new categories, timelines (published 21-Sep-2023)
Read Article on RAPS.org

FDA’s Califf: Expect to see more RWE-based regulatory decisions (published 22-Sep-2023)
Read Article on RAPS.org

FDA will continue to use remote inspection tools to assess pending applications (published 22-Sep-2023)
Read Article on RAPS.org

FDA’s IT strategy will focus on modernization, AI (published 22-Sep-2023)
Read Article on RAPS.org

CDER chief touts AI potential to reduce quality defects, encourages adoption of AI tools (published 26-Sep-2023)
Read Article on RAPS.org

CBER revises internal procedures for processing clinical holds, NDAs and BLAs (published 28-Sep-2023)
Read Article on RAPS.org

EFPIA: EU pharma manufacturing would ‘grind to a halt’ under proposed PFAS ban (published 28-Sep-2023)
Read Article on RAPS.org

FDA official discusses use of RWE in cancer research and approvals (published 29-Sep-2023)
Read Article on RAPS.org

• Review the recording from our webinar: Future Trends on Europeans Regulations on Medicines
• Learn how Veristat experts can help you better plan your decentralized clinical trials: Adopting Decentralized Clinical Trials
• Read our infographic to learn more about the different types of meetings under BsUFA FDA Meeting Types for Biosimilar Products
• Download the Infographic explaining the Types of FDA Meetings with CDER and CBER