FDA News - August 2017

JHOP Web Exclusives - FDA News & Updates, Online Only

A brief overview of cancer therapies approved by the FDA between June 1, 2017, and July 30, 2017.

In This Article


Darzalex Combined with Pomalidomide and Dexamethasone Approved for Relapsed/Refractory Multiple Myeloma

On June 16, 2017, the FDA approved daratumumab (Darzalex; Janssen Biotech), in combination with pomalidomide (Pomalyst) and dexamethasone, for the treatment of patients with multiple myeloma who have received at least 2 previous therapies, including lenalidomide (Rev­limid) and a proteasome inhibitor.

“This offers another alternative to patients with multiple myeloma who haven’t seen lasting effects from other types of treatment,” said Jan van de Winkel, PhD, Chief Executive Officer, Genmab.

The approval was based on data from the phase 1 EQUULEUS clinical trial involving 103 patients with multiple myeloma who received previous therapy. Patients received 16 mg/kg of daratumumab plus pomalidomide and dexamethasone until disease progression. The primary efficacy end point was the overall response rate (ORR).

The ORR was 59.2%. The stringent complete response rate was 7.8%, the complete response rate was 5.8%, the very good partial response rate was 28.2%, and the partial response rate was 17.5%.

The most common (≥20%) all-grade adverse reactions in patients who received daratumumab plus pom­alidomide and dexamethasone included infusion reactions, diarrhea, constipation, nausea, vomiting, fatigue, pyrexia, upper respiratory tract infection, muscle spasms, back pain, arthralgia, dizziness, insomnia, cough, and dyspnea. Overall, 49% of patients had serious adverse reactions, and 13% of patients discontinued therapy because of adverse reactions.

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Rituximab Combination Now Approved in 3 Blood Cancers

On June 22, 2017, the FDA approved the combination of rituximab and hyaluronidase human (Rituxan Hycela; Genentech) for the treatment of adults with follicular lymphoma, diffuse large B-cell lymphoma (DLBCL), or chronic lymphocytic leukemia (CLL).

Specifically, this approval covers indications that were previously approved for rituximab alone, including patients with previously untreated or nonprogressing relapsed or refractory follicular lymphoma; previously untreated DL­BCL; and previously treated and untreated CLL.

This approval gives patients a subcutaneous route for receiving rituximab that shortens its administration time by 5 to 7 minutes (compared with the several hours that an intravenous infusion can take) and provides the option of flat dosing.

The FDA’s approval was based on multiple randomized clinical trials that demonstrated the noninferiority of rituximab trough concentration levels in the combination therapy to those of intravenous rituximab, as well as the comparable efficacy and safety of the combination therapy and rituximab monotherapy.

In patients with follicular lymphoma, the most common (≥20%) adverse events associated with the combination therapy include infections, neutropenia, nausea, constipation, cough, and fatigue. For patients with DLBCL, the most common (≥20%) adverse events include infections, neutropenia, alopecia, nausea, and anemia. In patients with CLL, the most common (≥20%) adverse events are infections, neutropenia, nausea, thrombocytopenia, pyrexia, vomiting, and injection-site erythema.

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First FDA-Approved Test to Help Detect Several Leukemias and Lymphomas

On June 29, 2017, the FDA authorized the marketing of ClearLLab Reagents (from Beckman Coulter, Inc), the first test that is agency approved to aid flow cytometry in the marking and detecting of several blood cancers, including chronic leukemia, acute leukemia, non-Hodgkin lymphoma, myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms.

The ClearLLab test, which uses a fluorescent dye to mark proteins on the surface of cells for further analysis with a flow cytometer, identifies cancerous cells in blood, bone marrow, and lymph nodes, and gives clinicians information about the type of leukemia or lymphoma that is present.

“This represents a major step forward for the hematology-oncology community. Laboratories and health care professionals now have access to an FDA-validated test that provides consistent results to aid in the diagnoses of these serious cancers,” said Alberto Gutierrez, PhD, Director of the FDA Center for Devices and Radiological Health.

The ClearLLab test approval was based on a study showing that the ClearLLab test’s results accurately detected the presence of cancer 84.2% of the time.

The FDA notes that ClearLLab Reagents test results must be reviewed by trained professionals.

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DigniCap Cooling System Receives Expanded Indication for Use in Patients with Solid Tumors

On July 3, 2017, the FDA approved an expanded indication for the DigniCap Cooling System (from Dignitana) —a cooling cap used to reduce alopecia associated with chemotherapy—for patients with solid tumors. This expanded indication was approved based on peer-reviewed articles submitted by the manufacturer that examined use of the DigniCap in patients with solid tumors in areas of their body other than the breast.

“We are pleased to expand the use of this product for cancer patients with solid tumors to potentially minimize chemotherapy-induced hair loss,” said Binita Ashar, MD, Director of the FDA Center for Devices and Radiological Health.

The DigniCap was previously approved by the FDA in 2015 after trials in women with stage I or II breast cancer demonstrated the cap’s ability to lessen the severity of hair loss during treatment with alopecia-­inducing chemotherapy.

The most common adverse events associated with the DigniCap include cold-induced headaches, neck and shoulder discomfort, and chills and pain associated with extended wearing of the cooling cap.

The DigniCap is contraindicated in pediatric patients, those with certain cancers, and in patients receiving specific types of chemotherapy; it may not be suitable for patients with sensitivity to cold or who are susceptible to cold-related injuries.

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Blincyto Receives Expanded Indication to Include Patients with Ph+ B-Cell Precursor ALL

On July 11, 2017, the FDA approved an expanded indication for blinatumomab (Blincyto; Amgen) to include the treatment of patients with Philadelphia chromosome (Ph)-positive (Ph+), relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

This current regular FDA approval also provides confirmation of the 2014 accelerated approval granted to blinatumomab for the treatment of patients with Ph-negative relapsed or refractory B-cell precursor ALL. It also expands the indication to include all patients with relapsed or refractory B-cell precursor ALL, regardless of their Ph chromosome status.

The study that confirmed the initial approval was based on TOWER, a randomized clinical trial that compared blinatumomab and standard-of-care chemotherapy in 405 patients with relapsed or refractory B-cell precursor ALL. Results showed significant improvement in overall survival (OS) with blinatumomab versus chemotherapy (hazard ratio, 0.71; P = .012); the estimated median OS was 7.7 months versus 4.0 months, respectively.

The expanded indication to patients with Ph+ disease was based on ALCANTARA, a single-arm multicenter study of 45 patients with Ph+ relapsed or refractory with relapsed or refractory B-cell precursor ALL. Overall, 36% of patients reached complete remission with blinatumomab, with a median remission duration of 6.7 months.

The 2 studies did not report any new major adverse events.

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First CAR T-Cell Therapy Unanimously Recommended by FDA Advisory Committee for Treatment of B-Cell ALL in Children and Young Adults

On July 12, 2017, the FDA’s Oncologic Drugs Advisory Committee unanimously recommended the approval of Novartis’s CTL019 (tisagenlecleucel)—the first ever chimeric antigen receptor (CAR) T-cell therapy to be presented to the FDA for review for the treatment of pediatric patients and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).

The committee’s recommendation was based on a review of ELIANA, the first pediatric global CAR T-cell therapy clinical trial. The results from a multicenter trial and a single-center trial that assessed the drug’s safety and efficacy in pediatric patients and young adults with relapsed or refractory B-cell ALL also supported the recommendation.

Among the 63 patients who received CTL019 between April 2015 and August 2016, 63 (82.5%) patients experienced diseased remission, and 11 patients died.

“It’s a new world, an exciting therapy,” said Gwen Nichols, MD, Chief Medical Officer of the Leukemia & Lymphoma Society, which funded some of the research related to this treatment.

The FDA agreed to review the manufacturer’s Biologics License Application under its priority review status; if approved, CTL019 would become the first CAR T-cell therapy in the United States.

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Neratinib First Extended Adjuvant Treatment for Patients with Early-Stage, HER2-Positive Breast Cancer

On July 17, 2017, the FDA approved neratinib (Ner­lynx; Puma Biotechnology) for the long-term, adjuvant treatment of adults with early-stage, HER2-positive breast cancer who have received therapy that includes trastuzumab. Neratinib, a kinase inhibitor that blocks several cell-growth–promoting enzymes, is taken after treatment to lower the risk for cancer recurrence.

“HER2-positive breast cancers are aggressive tumors and can spread to other parts of the body, making adjuvant therapy an important part of the treatment plan. Now, these patients have an option after initial treatment that may help keep the cancer from coming back,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.

The approval of neratinib was based on the safety and efficacy results of a randomized clinical trial of 2840 patients with early-stage, HER2-positive breast cancer who had completed trastuzumab therapy in the past 2 years. Efficacy measures included the time to disease relapse or death from any cause. At 2 years, 94.2% of patients who received ner­atinib were alive and with no cancer recurrence versus 91.9% of patients who received placebo.

The common adverse events associated with neratinib include diarrhea, nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, muscle spasms, dyspepsia, liver damage, nail disorder, dry skin, distention, weight loss, and urinary tract infection.

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Last modified: August 11, 2017