FDA News: November 25th To December 18th

TOP - January 2021 Vol 14, No 1 - FDA Updates

This section provides a brief overview of new cancer drugs approved by the FDA between November 25, 2020, and December 18, 2020.

Orgovyx First Oral Hormone Therapy Approved for Advanced Prostate Cancer

On December 18, 2020, the FDA approved the oral gonadotropin-releasing hormone (GnRH) receptor antagonist, relugolix (Orgovyx; Myovant Sciences), for the treatment of adult patients with advanced prostate cancer. As an GnRH receptor, relugolix reduces production of testicular testosterone, a hormone known to stimulate the growth of prostate cancer.

“Today’s approval marks the first oral drug in this class, and it may eliminate some patients’ need to visit the clinic for treatments that require administration by a healthcare provider,” Richard Pazdur, MD, Director of FDA’s Oncology Center of Excellence, said in a press release. “This potential to reduce clinic visits can be especially beneficial in helping patients with cancer stay home and avoid exposure during the coronavirus pandemic.”

The approval of relugolix for this indication was based, in part, on results from a phase 3 clinical trial of men with advanced prostate cancer. Patients in this trial were randomized to relugolix once daily or injections of the hormone-targeting drug leuprolide every 3 months for 48 weeks. The objective of the study was to determine whether relugolix achieved and maintained low enough levels of testosterone (castrate levels) by day 29 through the end of the treatment course. In the 622 patients in the relugolix arm, the castration rate was 96.7%.

The most common (≥10%) adverse reactions reported among relugolix-treated patients included hot flush (54%), musculoskeletal pain (30%), fatigue (26%), constipation (12%), and diarrhea (12%).

The FDA previously granted relugolix a priority review for this indication.

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FDA Approves First Prostate-Specific Membrane Antigen–Targeted PET Imaging Drug for Prostate Cancer

On December 1, 2020, the FDA approved Gallium 68 PSMA-11 (Ga 68 PSMA-11; University of California), the first drug for positron emission tomography (PET) imaging of prostate-specific membrane antigen (PSMA)-positive lesions in men with prostate cancer. Ga 68 PSMA-11 is indicated for men with suspected metastatic prostate cancer that may be curable by surgery or by radiation, and for suspected recurrent prostate cancer based on elevated serum prostate-specific antigen (PSA) levels.

Ga 68 PSMA-11 is a radioactive drug that is administered via intravenous injection. Ga 68 PSMA-11 binds to PSMA, an important target for prostate cancer imaging.

The approval of Ga 68 PSMA-11 was based on the results of 2 prospective clinical trials in 960 men with prostate cancer who received 1 Ga 68 PSMA-11 with PET and computed tomography or with PET and magnetic resonance imaging. In the first study, 325 patients were candidates for surgery or had an increased risk for metastasis. Among the patients who had surgery, positive readings in the pelvic lymph nodes on Ga 68 PSMA-11 PET had a clinically significant rate of metastatic cancer confirmed by surgical pathology.

The second study included 635 patients with biochemical evidence of recurrent prostate cancer. In all, 74% of the patients had ≥1 positive lesions, as detected by Ga 68 PSMA-11 PET in at least 1 body region. Of those with positive Ga 68 PSMA-11 PET readings and correlative tissue pathology from biopsies, the results from baseline or follow-up imaging by conventional methods, and the serial PSA levels, 91% had confirmed local recurrence or metastasis of prostate cancer.

No serious adverse events were seen with Ga 68 PSMA-11. The most common adverse reactions were nausea, diarrhea, and dizziness. This drug is associated with a risk for misdiagnosis, because Ga 68 PSMA-11 binding may occur in other types of cancer, as well as certain nonmalignant processes.

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Danyelza Receives FDA Approval for High-Risk Neuroblastoma in the Bone and Bone Marrow

On November 25, 2020, the FDA granted accelerated approval to naxitamab-gqgk (Danyelza; Y-mAbs Therapeutics), a GD2-binding monoclonal antibody, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of relapsed or refractory high-risk neuroblastoma in the bone or bone marrow in pediatric patients aged ≥1 year and adults who achieved a partial response, minor response, or stable disease after receiving previous therapy. The FDA granted naxitamab priority review and breakthrough therapy, orphan drug, and rare pediatric disease designations.

“We believe that Danyelza in combination with GM-CSF is a much-needed treatment for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow who have historically not had approved treatments available,” said Claus Moller, Chief Executive Officer, Y-mAbs Therapeutics.

The approval of naxitamab was based on the results of 2 single-arm, open-label clinical trials in patients with relapsed or refractory neuroblastoma in the bone or bone marrow. The patients received naxitamab 3 mg/kg via intravenous infusion on days 1, 3, and 5 of each 4-week cycle in combination with GM-CSF subcutaneously at 250 µg/m2 daily on days –4 to 0 and at 500 µg/m2/day on days 1 to 5. The cycles were repeated every 4 to 8 weeks. At the investigator’s discretion, the patients received preplanned radiation to the main disease site in Study 201 and radiation therapy to nontargeted bony lesions or soft-tissue disease in Study 12-230.

The primary end points were overall response rate (ORR) per the revised International Neuroblastoma Response Criteria, and the duration of response (DOR). The ORR in Study 201 (N = 22) was 45% (95% confidence interval [CI], 24%-68%), and 30% of responders had a DOR of ≥6 months. In Study 12-230 (N = 38), the ORR was 34% (95% CI, 20%-51%), with a DOR of ≥6 months in 23% of responders. The responses were in the bone, the bone marrow, or in both.

The most common (≥25%) adverse reactions with naxitamab were infusion-related reactions, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection-site reaction, edema, anxiety, localized edema, and irritability.

Grade 3 or 4 adverse events (≥5%) were decreased levels of lymphocyte count, neutrophil count, hemoglobin, platelet count, potassium, glucose, calcium, albumin, sodium, and phosphate; and increased alanine aminotransferase.

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Last modified: July 22, 2021