Amgen’s Lumakras Obtains Early FDA Approval for KRAS-Mutated NSCLC
At the end of May, the U.S. Food and Drug Administration (FDA) approved Amgen’s Lumakras (sotorasib) as the first treatment for adult patients with non-small cell lung cancer (NSCLC) whose tumors have a KRAS G12C genetic mutation and who have received at least one prior systemic therapy. The application was approved two and half months earlier than the planned FDA action date.
- Lumakras is the first targeted therapy for cancers with any KRAS mutation, which accounts for about 25% of mutations in NSCLC, while KRAS G12C mutations make up about 13% of mutations.
- Approval was based on a study of 124 patients with locally advanced or metastatic KRAS G12C-mutated NSCLC with disease progression after receiving an immune checkpoint inhibitor and/or platinum-based chemotherapy. In the trial, 960 mg administered orally once-daily resulted in an objective response rate of 36% (proportion of patients whose tumor was destroyed or reduced). In addition, the median duration of response was 10 months.
- The most common side effects include diarrhea, musculoskeletal pain, nausea, fatigue, liver damage and cough. Serious liver damage and lung scarring and inflammation may also occur.
- The FDA granted the Amgen application Fast Track, Priority Review, Breakthrough Therapy and Orphan Drug designations. Lumakras is also being studied in multiple other solid tumors.
Myfembree is First Once-Daily Option for Heavy Bleeding with Fibroids
The FDA has approved once-daily oral Myfembree (relugolix, estradiol and norethindrone acetate), the first once-daily treatment for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months.
- Myfembree, from Myovant and Pfizer, is an oral gonadotropin-releasing hormone (GnRH) receptor antagonist, estrogen, and progestin combination. It is expected to be commercially available in June 2021.
- In the Phase 3 LIBERTY studies at Week 24, 72.1% and 71.2% of women receiving Myfembree achieved the preset endpoints compared to 16.8% and 14.7% of women in the placebo groups, respectively. Women receiving Myfembree experienced 82% and 84.3% reductions in menstrual blood loss when compared to the start of the study.
- Side effects included hot flush, abnormal uterine bleeding, alopecia (hair loss), and decreased libido (sex drive). The product label carries a boxed warning for thrombotic or thromboembolic disorders.
- Myfembree treatment is limited to 24 months due to the risk of bone loss which may not be reversible.
Camcevi Depot Formulation Cleared for Prostate Cancer
In May, the FDA approved Camcevi (leuprolide mesylate) as a new, long-acting option for advanced prostate cancer in adults. Camcevi, a gonadotropin releasing hormone (GnRH) agonist, is given as a 42 mg subcutaneous depot formulation injected every 6 months.
- In a Phase 3 study in 137 patients, the primary endpoint — the percentage of patients with suppressed serum testosterone (≤50 ng/dl) by day 28 and from day 28 to day 336 — was met in 97% of participants. Mean testosterone concentration was suppressed below castrate levels to 17.6 ng/dL on day 28.
- The most common (≥10%) side effects were hot flush, hypertension (high blood pressure), injection site reactions, upper respiratory tract infections, musculoskeletal pain, fatigue, and pain in extremity.
- Tumor flare due to elevated testosterone in the first weeks of treatment may lead to transient bone pain, ureteral obstruction, spinal cord compression and other symptoms.
Rybrevant is First Targeted Drug for NSCLC with Certain Genetic Mutations
This past month the FDA granted accelerated approval to Janssen’s Rybrevant (amivantamab-vmjw) to treat adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test. Eligible patients have also progressed on or after platinum-based chemotherapy.
- Rybrevant is the first fully-human, bispecific antibody approved for the treatment of patients with NSCLC that targets EGFR exon 20 insertion mutations.
- It is given as an intravenous (IV) infusion weekly for four weeks, then every two weeks. Premedication is recommended to reduce the risk of infusion-related reactions.
- In the ongoing Phase 1 CHRYSALIS study, the confirmed overall response rate was 40%, with 3.7% of patients having a complete response and 36% achieving a partial response.
- Rybrevant may cause serious side effects such as infusion-related reactions, interstitial lung disease/pneumonitis, skin reactions, and eye problems. Common side effects include rash, musculoskeletal pain, shortness of breath, and nausea, among others.
Empaveli: First C3 Inhibitor Granted Approval for Rare Blood Disorder
FDA has approved Apellis Pharmaceuticals’ Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, genetic, life-threatening blood disorder that leads to red blood cell destruction and anemia. Empaveli is the first PNH treatment that binds to and inhibits complement protein C3, a part of the immune defense system.
- Approval was based on the 16-week, head-to-head PEGASUS study, which compared Empaveli to Soliris (eculizumab) in 80 adults with PNH. Empaveli is given by subcutaneous infusion twice weekly.
- Empaveli met the primary endpoint of superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin.
- Empaveli carries a boxed warning and is available only through a Risk Evaluation and Mitigation Strategy (REMS) due to increased risk of meningococcal and other serious infections caused by encapsulated bacteria.
- Common side effects include injection-site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue.
Posted: May 2021
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