FDA Approvals: LYNPARZA, BESPONSA – Drug and Device Digest

FDA BRIEF: Week of  August 14, 2017

FDA approved

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LYNPARZA (olaparib) tablets


INDICATION FOR USE: Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy

ADDRESSING UNMET NEED: Addition of new indication and new formulation (tablet)


  • Initial approval of capsule formulation in 2014 for the treatment of BRCA-mutated advanced ovarian cancer
  • New tablet formulation also approved for this indicaiton and new indication
  • Tablets and capsules are not interchangeable; capsules will be phased out of market and will be available only through Lynparza Specialty Pharmacy Network
  • Postmarketing commitments:  progression-free survival (PFS) and molecular characteristics,  overall response rate (ORR) and duration of response (DOR) from ongoing clinical studies 

MECHANISM OF ACTION: Inhibitor of poly (ADP-ribose) polymerase (PARP) enzymes that are involved in DNA transcription and DNA repair


  • Two randomized, placebo-controlled, double-blind, multicenter studies, n=295, 265, patients with recurrent ovarian cancers who were in response to platinum-based therapy
  • Major efficacy outcome: Investigator-assessed PFS evaluated according to RECIST, version 1.1.
  • Study 1 (recurrent germline BRCA-mutated cancer): Estimated median PFS was 19.1 vs. 5.5 months (olaparib vs. placebo),  p<0.0001)
  • Study 2 (regardless of BRCA status):  Median PFS was 8.4 months vs. 4.8 months, p<0.0001


  • Most common adverse reactions: Anemia, nausea, fatigue (including asthenia), vomiting, nasopharyngitis, diarrhea, arthralgia/myalgia, dysgeusia, headache, dyspepsia, decreased appetite, constipation, and stomatitis
  • Most common laboratory abnormalities: Decrease in hemoglobin, increase in mean corpuscular volume, decrease in lymphocytes, decrease in leukocytes, decrease in absolute neutrophil count, increase in serum creatinine, and decrease in platelets


Image for trademark with serial number 86916876

BESPONSA (inotuzumab ozogamicin) for injection


INDICATION:  Treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)


  • 5,970 in United States will be diagnosed with ALL in 2017; ~1,440 will die from disease
  • Low life expectancy for B-cell ALL patients with nonresponsive / relapsed cancer
  • New, targeted treatment option

REG PATHWAY: BLA, Orphan drug designation, Priority Review, Breakthrough Therapy

  • Postmarketing requirements: Characterize toxicity after hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients, randomized trial in patients with relapsed or refractory acute lymphoblastic leukemia, Bioburden and endotoxin test method qualification

MECHANISM OF ACTION:  CD22-directed antibody-drug conjugate (ADC), is a cytotoxic agent covalently attached to antibody via linker. Anticancer activity due to binding of the ADC to CD22-expressing tumor cells


  • Randomized (1:1), open-label, international, multicenter study in patients with relapsed or refractory ALL, n= 326, BESPONSA vs.  Investigator’s choice of chemotherapy
  • Efficacy: Complete Response (CR), duration of remission (DoR) and Overall Survival



  • Boxed warning: Severe liver damage (hepatotoxicity), including blockage of veins in the liver
  • Other serious side effects: Myelosuppression, infusion-related reactions, QT interval prolongation, reproductive toxicity
  • Common side effects:  Thrombocytopenia, neutropenia, leukopenia, infection, anemia, fatigue, hemorrhage, pyrexia, nausea, headache, febrile neutropenia, liver damage, abdominal pain, hyperbilirubinemia


Image credit: AstraZeneca, Pfizer

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