Monthly News Roundup - August 2023
Medically reviewed by Drugs.com.
FDA Approves Zurzuvae, First Oral Treatment for Postpartum Depression
In August, the U.S. Food and Drug Administration (FDA) cleared Zurzuvae (zuranolone) from Sage Therapeutics / Biogen. Zurzuvae is the first FDA-approved oral agent to treat postpartum depression (PPD) in adults. It is given as a once-daily treatment for 14 days.
- Zurzuvae is a neuroactive steroid (NAS) GABA-A receptor positive allosteric modulator (PAM). GABA is the major inhibitor signaling pathway in the central nervous system and helps regulate brain function.
- Approval for PPD was based on results from two Phase 3 clinical trials. In the SKYLARK study, treatment with Zurzuvae 50 mg once daily significantly improved symptoms of PPD at Day 15 (HAMD-17 total score) - and as early as Day 3 - with a sustained effect to Day 45. HAMD-17 is a common measure of depression severity.
- The label carries a Boxed Warning for driving impairment or engaging in hazardous activities due to drowsiness (somnolence). Patients should not drive or engage in hazardous activities until at least 12 hours after Zurzuvae administration for the entire 14-day treatment course. The most common (>5%) side effects were somnolence, dizziness, diarrhea, fatigue and urinary tract infection (UTI).
- Following DEA controlled substance scheduling, Zurzuvae is expected to launch in the U.S. in the fourth quarter of 2023.
Higher-Dose Eylea HD Approved for Retinal Disorders of the Eye
Eylea HD (aflibercept 8 mg injection) is a higher dose formulation of the approved vascular endothelial growth factor (VEGF) inhibitor aflibercept 2 mg injection (Eylea). It’s indicated to treat patients with neovascular (wet) age-related macular degeneration (nAMD), diabetic macular edema (DME), and diabetic retinopathy (DR).
- Approval of Regeneron’s Eylea HD was based on the results of two 48-week long studies, PULSAR and PHOTON with over 1,600 patients with nAMD or DME. These studies evaluated Eylea HD (8 mg) compared to Eylea injection (2 mg). Both studies found that Eylea HD effectiveness was similar to Eylea (noninferior), with similar vision gains at 48 weeks for both 12- and 16-week dosing regimens after 3 initial monthly doses, compared to an Eylea 8-week dosing regimen after initial doses. Most people receiving Eylea HD were able to stay on their 12- or 16-week dosing regimens.
- Eylea HD is given by intravitreal (into the back of the eye) injection. Your retinal eye specialist will wash and numb your eye before the injection and give you antibiotic eye drops. You may feel some pressure while you are receiving the injection but it should not be painful.
- Side effects with Eylea HD include: cataract, increased redness in the eye, increased pressure in the eye, eye discomfort, pain, or irritation, blurred vision, vitreous (gel-like substance) floaters, vitreous detachment, injury to the outer layer of the eye, and bleeding in the back of the eye.
FDA Grants Accelerated Approval of Elrexfio for Relapsed or Refractory Multiple Myeloma
In August, Pfizer announced the accelerated approval of Elrexfio (elranatamab-bcmm), a B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb), for the treatment of adults with multiple myeloma that came back or stopped responding to treatment, and who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. It works by binding to BCMA on myeloma cells and CD3 on T-cells, bringing them together to activate T-cells and cause cancer cell death.
- Elrexfio is the first BCMA-directed therapy with every 2 week dosing for responding patients (after 24 weeks of weekly therapy initially). It comes as a ready-to-use fixed dose subcutaneous (under the skin) injection and is given until disease progression or unacceptable toxicity.
- Approval was based on the single arm, Phase 2 MagnetisMM-3 study which demonstrated clinically meaningful response rates and duration of response among heavily pretreated RRMM patients who received Elrexfio as their first BCMA-directed therapy. Among the patients in this study who received four or more lines of therapy prior to Elrexfio, the overall response rate was 58%, with an estimated 82% maintaining the response for at least 9 months. The median time to first response was 1.2 months.
- The label carries a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity (NT), including immune effector cell-associated neurotoxicity syndrome (ICANS). Warnings include infections, neutropenia, hepatotoxicity and embryo-fetal toxicity. The most common side effects of Elrexfio (at least 20% incidence) include CRS, fatigue, injection site reaction, diarrhea, and upper respiratory tract infection, among others.
FDA Approves Tyruko, the First Biosimilar for Treating Relapsing MS
This past month the FDA cleared Tyruko (natalizumab-sztn), an integrin receptor antagonist biosimilar to Tysabri, as the first biosimilar to treat adults with relapsing forms of multiple sclerosis. Tyruko, from Sandoz, is also used to treat Crohn’s Disease (CD) in adults. It is the first biosimilar to Tysabri (natalizumab).
- A biosimilar is a biological product that is highly similar to a reference biologic (in this case Tysabri) and for which there are no clinically meaningful differences in terms of safety, purity, and potency. A biosimilar is not considered a “generic” because they are made from living cells. Biosimilars may help to lower overall costs for patients and the healthcare system.
- Approval of Tyruko was based on evidence showing no clinically meaningful differences in terms of safety, purity and potency to the reference product Tysabri.
- The label contains a Boxed Warning about the increased risk of progressive multifocal leukoencephalopathy (PML), a viral infection of the brain that usually leads to death or severe disability. Due to PML risk, Tyruko is only available through a restricted drug distribution program known as a REMS.
- Additional warnings include risks regarding herpes infections, thrombocytopenia (low blood platelet count), immunosuppression (an increased risk of certain infections), serious hypersensitivity reactions such as anaphylaxis and hepatotoxicity (significant liver injury).
- The most common side effects associated with natalizumab products are headache and fatigue, arthralgia (joint pain), urinary tract infection, lower respiratory tract infection, gastroenteritis (stomach flu), vaginitis (infection or inflammation of the vagina), depression, pain in extremity, abdominal (stomach area) discomfort, diarrhea and rash.
FDA Approves Akeega for BRCA-Positive Metastatic Castration-Resistant Prostate Cancer (mCRPC)
Prostate cancer is one of the most common cancers in the U.S., with an estimated 288,300 new cases and nearly 35,000 deaths expected in 2023. In response, the FDA has approved Akeega, a once daily oral dual action tablet (DAT) combining a PARP inhibitor (niraparib) with abiraterone acetate, given with prednisone, to treat adults with deleterious or suspected deleterious BRCA-positive metastatic castration-resistant prostate cancer (mCRPC), as determined by testing.
- Niraparib is a Poly (ADP-ribose) Polymerase (PARP) inhibitor, a targeted treatment that stops cancer cells from repairing and leads to cell death and a reduction in tumor growth. Abiraterone acetate is an androgen biosynthesis inhibitor.
- Approval was based on the Phase 3 MAGNITUDE study, where BRCA-positive patients treated with Akeega plus prednisone showed a statistically significant 47% risk reduction for radiographic progression-free survival (rPFS).
- The recommended adult dosage of Akeega is 200 mg niraparib / 1,000 mg abiraterone acetate orally once daily on an empty stomach in combination with 10 mg prednisone daily. Do not eat food two hours before and one hour after taking Akeega.
- Patients normally continue treatment until disease progression or unacceptable toxicity. Lower strength Akeega (50 mg niraparib / 500 mg abiraterone acetate) is available for dose adjustments due to adverse effects.
- Warnings include Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), myelosuppression (low blood cell counts), liver toxicity, low potassium, low blood sugar, fluid retention and heart side effects, among others.
- The most common side effects in at least 20% of patients who received Akeega plus prednisone were musculoskeletal pain, fatigue (tiredness), constipation, hypertension (high blood pressure) and nausea.
Abrysvo is First Maternal Vaccine Approved to Help Protect Newborns From RSV
In August, Pfizer’s Abrysvo (respiratory syncytial virus vaccine) received an expanded use for active immunization of pregnant individuals at 32 through 36 weeks gestational age for the prevention of lower respiratory tract disease (LRTD) and severe LRTD caused by respiratory syncytial virus (RSV) in infants from birth through 6 months of age.
- RSV is a contagious virus and a common cause of respiratory illness. It can lead to severe illness in young infants, older adults, and individuals with certain chronic medical conditions.
- Approval was based on the Phase 3 MATISSE placebo-controlled study, which evaluated the efficacy, safety, and immunogenicity of the vaccine. Efficacy results met the statistical criterion for success (a CI lower bound >20%) for reducing severe LRTD due to RSV, at all timepoints to within 180 days, but did not meet criterion for reducing LRTD due to RSV; however, clinically meaningful effectiveness was seen after 90 days through 180 days after birth.
- Abrysvo is composed of two prefusion F (preF) proteins selected to optimize protection against RSV A and B strains. Prefusion F is a key form of the viral fusion protein (F) that RSV uses to enter human cells. It is given as a single intramuscular (IM) injection.
- Warnings and precautions associated with Abrysvo include the potential risk of preterm birth; it is administered at 32 to 36 weeks gestational age to help prevent this risk. Common side effects (≥10%) in pregnant individuals include pain at the injection site (40.6%), headache (31%), muscle pain (26.5%), and nausea (20%).
- In May 2023, Abrysvo was first approved for the prevention of LRTD caused by RSV in people 60 years of age and older.
Ingrezza Label Expanded for the Treatment of Chorea Associated With Huntington's Disease
This past month the FDA approved a new use for Ingrezza (valbenazine) - to treat adults with chorea associated with Huntington's disease (HD). In this condition, patients have involuntary, irregular and unpredictable movements that may affect walking, swallowing and speech. Ingrezza does not treat other symptoms of Huntington’s disease, such as thinking or emotional problems.
- Ingrezza is a highly selective vesicular monoamine transporter 2 (VMAT2) inhibitor. It is taken by mouth once a day, with or without food. It is available in 40 mg, 60 mg and 80 mg capsules. Your doctor will determine your specific dose.
- Approval was based on results from the KINECT-HD studies that showed a significant 3-fold greater improvement in chorea severity compared to placebo over 12 weeks (chorea severity score improvement: 4.6 points vs 1.4 points, respectively). Chorea severity was reduced by about 40% from baseline (the start of the study), and about 50% of patients saw at least a 40% reduction in severity by week 12.
- The most common side effects (≥5%) reported with Ingrezza in studies of chorea associated with Huntington’s disease are sleepiness, lack of energy, urticaria (hives), rash, and insomnia (trouble sleeping).
- Ingrezza, from Neurocrine Biosciences, was also approved for the treatment of adults with tardive dyskinesia in April 2017.
Reblozyl Cleared as First-Line Treatment of Anemia in Myelodysplastic Syndromes (MDS)
In August, the FDA expanded the use for Reblozyl (luspatercept-aamt) from Bristol Myers Squibb to treat anemia (low red blood cells) in ESA-naïve adults (adults without previous erythropoiesis stimulating agent [ESA] use). ELigible patients exhibit very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions.
- Myelodysplastic syndromes are a group of blood cancers in which immature blood cells in the bone marrow (red blood cells, white blood cells and platelets) do not become healthy blood cells. Most patients with MDS experience ongoing anemia and require RBC transfusions that are associated with an increased risk of iron overload.
- Reblozyl works by increasing the number of RBCs in the blood by helping immature red blood cells grow, and this reduces the amount of blood transfusions required.
- Approval was based on the Phase 3 COMMANDS trial, which showed that a statistically significant 58.5% of patients treated with Reblozyl vs. 31.2% of patients treated with epoetin alfa achieved the primary endpoint of RBC transfusion independence (RBC-TI) of at least 12 weeks with a mean hemoglobin (Hb) increase of at least 1.5 g/dL within the first 24 weeks. Patients treated with Reblozyl had durable responses with nearly 2.5 years of RBC-TI ≥12 weeks (median).
- Reblozyl is given as a subcutaneous (under the skin) injection. The recommended dose is 1 mg/kg subcutaneously once every 3 weeks.
- The most common (>10%) side effects were diarrhea, fatigue (tiredness), hypertension (high blood pressure), peripheral edema (fluid retention in the extremities, like legs or arms), nausea, and dyspnea (shortness of breath).
Posted August 2023
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