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                Resources for Information | Approved Drugs

Resources for Information | Approved Drugs

                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
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                        Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description Home
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          Resources for Information | Approved Drugs
          FDA D.I.S.C.O. Burst Edition: FDA approvals of Imbruvica (ibrutinib) for pediatric patients with chronic graft versus host disease, and Pemazyre (pemigatinib) for adults with relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangementResources for Information | Approved Drugs FDA D.I.S.C.O. Burst Edition: FDA approvals of Imbruvica (ibrutinib) for pediatric patients with chronic graft versus host disease, and Pemazyre (pemigatinib) for adults with relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement
  
  	
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Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on two recent FDA cancer drug approvals. On August 24, 2022, the FDA approved ibrutinib (brand name Imbruvica) for pediatric patients 1 year of age or older with chronic graft versus host disease after failure of 1 or more lines of systemic therapy. Formulations include capsules, tablets, and oral suspension.

Efficacy was evaluated in iMAGINE, an open-label, multi-center, single-arm trial of ibrutinib for pediatric and young adult patients 1 year to less than 22 years old with moderate or severe chronic graft versus host disease. The trial included 47 patients who required additional therapy after failure of 1 or more lines of systemic therapy. Patients were excluded if single organ genitourinary involvement was the only manifestation of chronic graft versus host disease.

The median age of patients was 13 years, ranging from 1 to 19. Selected demographics of the 47 patients were as follows: 70% male, 36% White, 9% Black or African American, 55% other or unreported.

The main efficacy outcome measure was overall response rate through Week 25. Overall response rate included complete response or partial responses according to the 2014 NIH Consensus Development Project Response Criteria. Overall response rate by Week 25 was 60%. The median duration of response was 5.3 months. The median time from first response to death or new systemic therapies for chronic graft versus host disease was 14.8 months.

The most common adverse reactions occurring in more than 20% of patients, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

On August 26, 2022, the FDA approved pemigatinib (brand name Pemazyre) for adults with relapsed or refractory myeloid/lymphoid neoplasms with fibroblast growth factor receptor 1 rearrangement.

Efficacy was evaluated in FIGHT-203, a multicenter open-label, single-arm trial that included 28 patients with relapsed or refractory myeloid/lymphoid neoplasm with fibroblast growth factor receptor 1 rearrangement. Eligible patients were either not candidates for or have relapsed after allogeneic hematopoietic stem cell transplantation or after a disease modifying therapy, for example chemotherapy. Pemigatinib was administered until disease progression, unacceptable toxicity, or until patients were able to receive allogeneic hematopoietic stem cell transplantation.

Selected demographics and baseline characteristics were: median age of 65 years ranging from 39 to 78; 64% female; 68% White, 3.6% Black or African American, 11% Asian, and 3.6% American Indian/Alaska Native; and 88% ECOG performance status of 0 or 1.

Efficacy was established based on complete response rates per the response criteria relevant to the morphologic disease type. Of the 18 patients with chronic phase in the marrow with or without extramedullary disease, 14 achieved complete response. The median time-to- complete response was 104 days. The median duration was not reached. Of the 4 patients with blast phase in the marrow with or without extramedullary disease, 2 achieved complete response. Of 3 patients with extramedullary disease, only 1 achieved a complete response. For all 28 patients, including 3 patients without evidence of morphologic disease, the complete cytogenetic response rate was 79%.

The most common adverse reactions occurring in more than 20% of patients were hyperphosphatemia, nail toxicity, alopecia, stomatitis, diarrhea, dry eye, fatigue, rash, abdominal pain, anemia, constipation, dry mouth, epistaxis, serous retinal detachment, extremity pain, decreased appetite, dry skin, dyspepsia, back pain, nausea, blurred vision, peripheral edema, and dizziness.

The most common Grade 3 or 4 laboratory abnormalities occurring in more than 10% of patients were decreased phosphate, decreased lymphocytes, decreased leukocytes, decreased platelets, increased alanine aminotransferase, and decreased neutrophils.

Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatFDA.

Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.

Follow the Division of Drug Information on Twitter @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology.  Send your feedback to FDAOncology@fda.hhs.gov. Thanks for tuning in to the D.I.S.C.O. Burst Edition.

                                                                                  Content current as of:
      09/28/2022Resources for Information | Approved Drugs

          
                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
          Verified Clinical Benefit | Cancer Accelerated Approvals
          Withdrawn | Cancer Accelerated Approvals
          Other | Cancer Accelerated Approvals


                        Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description
 
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