Cell and gene therapy: FDA Approvals, Recalls, 510(k) Clearances and Side Effect Reports

• 3new drug approvals (since 1 Jan 2023)
• 4,744side effect reports (reports received since 1 Jan 2023)

Each number has its full source under its own list below.

• 23Dec2025

  Yartemlea (narsoplimab)

  Approved for: YARTEMLEA is indicated for the treatment of adult and pediatric patients 2 years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA…

  BLA761152
• 21Jan2025

  Grafapex (treosulfan)

  Approved for: GRAFAPEX is an alkylating drug indicated for: Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adu…

  NDA214759
• 8Sep2023

  Aphexda (motixafortide)

  Approved for: APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation i…

  NDA217159

Source: openFDA, Novel drug approvals (new molecular entities and new biologics), since 1 Jan 2023. Retrieved 12 Nov 2026.

1. 1Febrile neutropenia330 reports
2. 2Mucosal inflammation323 reports
3. 3Cytomegalovirus infection reactivation291 reports
4. 4Acute graft versus host disease in skin263 reports
5. 5Pyrexia255 reports
6. 6Venoocclusive liver disease186 reports
7. 7Acute graft versus host disease180 reports
8. 8Infection170 reports
9. 9Acute graft versus host disease in intestine156 reports
10. 10Diarrhoea147 reports

Anyone can send a report. A report does not prove that the medicine caused the reaction, and the counts do not show how often it happens.

Source: openFDA, FDA Adverse Event Reporting System (FAERS), reports by reaction, reports received since 1 Jan 2023. Retrieved 12 Nov 2026.

• Oncology
• Biologics and biosimilars