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Monthly News Roundup - September 2024

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Sep 30, 2024.

FDA Approves Ocrevus Zunovo, a Twice-A-Year Subcutaneous Injection for Relapsing and Progressive Multiple Sclerosis

In September, Genentech announced the approval of Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq), a subcutaneous (SC) injection for the treatment of adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease and primary progressive MS.

• Ocrevus Zunovo is a CD20-directed cytolytic antibody and hyaluronidase combination. Recombinant human hyaluronidase PH20 (rHuPH20), an enzyme, degrades hyaluronan (a sugar) in the area under the skin. This allows the medication to be rapidly dispersed and absorbed into the bloodstream when injected subcutaneously.
• Ocrevus Zunovo is given twice-a-year by a healthcare professional as a 10-minute subcutaneous (SC) injection in the stomach area (abdomen). Patients will still spend about one hour or more in the clinic due to premedications and monitoring for infusion reactions. With Ocrevus maintenance intravenous (IV) infusions, also given every 6 months, patients may spend at least 3.5 hours or longer in the clinic, depending upon infusion rate.
• Approval was based on the Phase III OCARINA II trial, which showed that Ocrevus Zunovo was consistent with the IV formulation (non-inferior), showing suppression of relapse activity (97%) and MRI lesions (97%) through 48 weeks. Over 92% of patients reported being satisfied or very satisfied with the SC administration of Ocrevus Zunovo.
• Warnings and precautions include injection reactions, infections, Progressive Multifocal Leukoencephalopathy (PML), reduction in immunoglobulins, increased risk of cancer and immune-mediated colitis.
• The most common adverse event was mild to moderate injection reactions and was more frequently reported with the first injection (49% of participants). No injection reactions led to treatment withdrawal.

AstraZeneca’s Tagrisso Approved to Treat Unresectable, Stage III EGFR-Mutated NSCLC

AstraZeneca has announced the FDA approval of Tagrisso (osimertinib) to treat adults locally advanced (has spread to nearby tissues), unresectable (unable to be removed by surgery), stage III non-small cell lung cancer (NSCLC) whose disease has not progressed during or following chemotherapy and radiation treatment and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test.

• Lung cancer is the leading cause of cancer death among both men and women, accounting for about 20% of all cancer deaths. About 10% to 15% of NSCLC patients in the US have EGFR mutations.
• Tagrisso is a tyrosine kinase inhibitor and targets the EGFR receptor, specifically the T790M mutation that blocks the signaling of EGFR to lead to cell death.
• Tagrisso tablets are taken orally once daily, with or without food, until disease progression or unacceptable toxicity.
• Approval was based on the Phase III LAURA trial. Patients were treated with Tagrisso 80 mg once daily oral tablets until disease progression, unacceptable toxicity or other discontinuation criteria were met. Tagrisso reduced the risk of disease progression or death by 84% compared to placebo (hazard ratio 0.16; 95% confidence interval 0.10-0.24; p<0.001. Median progression-free survival (PFS) was 39.1 months in patients treated with Tagrisso versus 5.6 months for placebo.
• Warnings and precautions include interstitial lung disease / pneumonitis; QTc interval prolongation; cardiomyopathy; keratitis; erythema multiforme major, Stevens-Johnson syndrome, and toxic epidermal necrolysis; cutaneous vasculitis; aplastic anemia; and embryo-fetal toxicity.
• Common adverse events included lymphopenia, leukopenia, ILD/pneumonitis, thrombocytopenia, neutropenia, rash, diarrhea, nail toxicity, musculoskeletal pain, cough, and COVID-19.

FDA Grants Full Approval to Filspari to Slow Kidney Function Decline in IgA Nephropathy

This past month Travere Therapeutics announced the full FDA approval for Filspari (sparsentan) to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. Filspari is classified as a Dual Endothelin Angiotensin Receptor Antagonist (DEARA).

• IgAN, also called Berger's disease, is a rare kidney disorder characterized by the buildup of immunoglobulin A (IgA) in the kidneys. Deposits of IgA in the kidneys cause a breakdown of the normal filtering mechanisms leading to proteinuria and hematuria (protein / blood in the urine), kidney pain, edema (fluid retention), and high blood pressure. IgAN is a leading cause of kidney failure due to glomerular disease, affecting up to 150,000 people in the U.S.
• Filspari is given as a once-daily, oral, non-immunosuppressive treatment. It is only available via a Risk Evaluation and Mitigation Strategy (REMS) due to the risks of liver injury and birth defects.
• Filspari was previously given FDA accelerated approval based on the surrogate marker of proteinuria in Feb. 2023. Full approval was based on 2-year confirmatory results from the head-to-head Phase 3 PROTECT Study. Based on 404 randomized patients, Filspari significantly slowed kidney function decline compared to irbesartan (-3.0 mL/min/1.73 m2/year for Filspari and -4.2 mL/min/1.73 m2/year for irbesartan). The reduction in proteinuria compared to irbesartan observed in week 36 was durable to the 2-year mark.
• Filspari carries a Boxed Warning for liver toxicity and embryo-fetal toxicity (harm to an unborn baby) and is only available via the REMS. Other warnings and precautions associated with Filspari include hypotension (low blood pressure), acute kidney injury, hyperkalemia (high potassium), and fluid retention.
• Common side effects (in at least 5% of patients) include hyperkalemia (high potassium levels), hypotension / low blood pressure (including orthostatic hypotension), peripheral edema (fluid build-up), dizziness, anemia (low red blood cell count), and acute kidney injury.

Johnson & Johnson’s Tremfya Approved for Adults with Ulcerative Colitis

In September, the FDA approved Johnson & Johnson’s Tremfya (guselkumab), an interleukin-23 (IL-23) blocker, for the treatment of adults with moderately to severely active ulcerative colitis (UC). Tremfya is also approved to treat adults with moderate to severe plaque psoriasis or active psoriatic arthritis.

• Ulcerative colitis (UC) is a chronic disease of the large intestine in which the lining of the colon becomes inflamed. Symptoms may include: diarrhea (possibly with blood or pus), stomach pain and cramping, weight loss, fever, fatigue, rectal pain, urgent need to defecate.
• Tremfya is a monoclonal antibody that blocks IL-23 while also binding to CD64, a receptor on cells that produce ILK-23. Interleukins are made by white blood cells. They help to fight infection in the body and regulate the immune system. These interleukin proteins are thought to contribute to long-lasting inflammation in conditions like plaque psoriasis, psoriatic arthritis and ulcerative colitis.
• For UC, Tremfya is given as an initial 200 mg intravenous (IV) induction dose by a healthcare provider at Week 0, Week 4, and Week 8. Then it’s given as a 100 mg subcutaneous (SC) maintenance injection at Week 16, and every 8 weeks thereafter; OR as a 200 mg SC injection at Week 12, and every 4 weeks thereafter. SC doses may be given by the patient or a caregiver, after training.
• Approval was based on the placebo-controlled Phase 2b/3 QUASAR study in 701 adult patients with UC. Patients had previously received conventional therapy, other biologics and/or JAK inhibitors without success or tolerance. Results showed that 50% of patients receiving Tremfya 200 mg subcutaneous (SC) maintenance every 4 weeks and 45% of patients receiving Tremfya 100 mg SC every 8 weeks achieved clinical remission at week 44 compared to 19% of placebo-treated patients (p<0.001). In addition, over one-third of patients achieved endoscopic remission at one year with Tremfya vs. 15% of placebo-treated patients (p<0.001).
• The most common adverse reactions during Induction (≥2%) were respiratory tract infections and during maintenance (≥3%) were injection site reactions, arthralgia (joint pain), and upper respiratory tract infections.

FDA Approves Miplyffa Capsules to Treat Ultra-Rare Niemann-Pick Disease Type C

The FDA has granted approval for Zevra Therapeutics’ Miplyffa (arimoclomol) to be used in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease Type C (NPC) in adult and pediatric patients 2 years of age and older.

• Niemann-Pick disease type C (NPC) is a rare genetic lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes. This results in an inability to transport cholesterol and other lipids into cells leading to tissue accumulation, including in the brain. Clinically, this can result in impaired speech, cognition (thinking), swallowing, walking, and fine motor skills.
• Miplyffa, a heat shock protein amplifier, is thought to work by amplifying the production of heat shock proteins to rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes, although the exact mechanism is unknown.
• Miplyffa capsules (47 mg, 62 mg, 93 mg and 124 mg) are administered orally 3 times a day, with or without food. Dosages are weight-based with adjustments needed in kidney impairment. For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment.
• Approval was based on a 12-month, randomized, placebo-controlled study in 50 patients aged 2 to 19 years. In the study, 76% of patients in the Miplyffa group and 81% of those in the placebo group received miglustat. Efficacy was measured with the R4DNPCCSS, a 4-item assessment of ambulation, speech, swallowing, and fine motor skills, with higher scores representing greater severity of disease.
• Miplyffa in combination with miglustat halted disease progression through 12 months of treatment, as demonstrated by a decrease of 0.2 points from baseline on the R4DNPCCSS compared to 1.9 points of progression for patients treated with miglustat alone. There was insufficient data to determine the effectiveness of Miplyffa without miglustat in patients with NPC.
• Warnings and precautions include: hypersensitivity (allergic) reactions, possible fetal harm, and increases in serum creatinine.
• The most common adverse reactions (incidence at least 15%) are upper respiratory tract infection, diarrhea, and decreased weight.

IntraBio’s Aqneursa Oral Suspension Cleared to Treat Niemann-Pick Disease Type C

The FDA has approved IntraBio’s Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children weighing at least 15 kg (33 lbs). Aqneursa is modified amino acid but the molecular target for levacetylleucine in the treatment of NPC is unknown.

• Niemann-Pick disease type C (NPC) is a rare genetic lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes. This results in an inability to transport cholesterol and other lipids into cells leading to tissue accumulation, including in the brain. Clinically, this can result in impaired speech, cognition (thinking), swallowing, walking, and fine motor skills.
• The recommended dosage for Aqneursa oral granule packets is based on the patient’s actual body weight (in kg). It is administered orally up to 3 times daily, taken with or without food. The granules are dissolved in water, orange juice or almond milk to form a suspension prior to administration.
• Aqneursa is the only FDA-approved stand-alone therapy indicated for the treatment of NPC. Approval was based on the 24-week IB1001-301 study, a placebo-controlled, randomized clinical trial with 60 patients 4 years and older with NPC. Efficacy was evaluated using the functional SARA (fSARA) tool that assesses gait, sitting, stance, and speech disturbance domains of the original SARA. The majority of the patients (85%) received miglustat treatment prior to randomization and during the trial.
• Results showed a statistically significant greater improvement in fSARA score with a mean treatment difference of -0.4 (95% CI: -0.7, -0.2), with Aqneursa scores of 5.1 and placebo scores of 6.5 at 24 weeks. Aqneursa significantly improved neurological signs and symptoms and demonstrated functional benefits within 12 weeks.
• Warnings and precautions include possible fetal harm. The most common adverse reactions (incidence at least 5% and greater than placebo) are abdominal (stomach area) pain, dysphagia (trouble swallowing), upper respiratory tract infections, and vomiting.

FDA Approves Oral Cobenfy for Schizophrenia, a First-In-Class Muscarinic Agonist

Bristol Myers Squibb has announced the approval of Cobenfy (xanomeline and trospium), an M1 / M4 muscarinic receptor agonist and antimuscarinic combination for the treatment of schizophrenia in adults. Schizophrenia is a potentially disabling mental illness that affects how a person thinks, feels and behaves. It is estimated to impact approximately 2.8 million people in the U.S.

• Cobenfy represents the first new class of medicine in over 30 years to treat schizophrenia by selectively targeting M 1 and M 4 receptors in the brain without blocking D2 receptors. Cobenfy labeling does not contain a Boxed Warning or atypical antipsychotic class warnings and precautions.
• The recommended starting dose is 50 mg / 20 mg orally twice daily for at least 2 days, then increase to 100 mg / 20 mg twice daily for at least 5 days. Dosage may be increased to 125 mg / 30 mg orally twice daily based on patient tolerability and response. Take at least 1 hour before a meal or at least 2 hours after a meal. Do not open capsules.
• Lower doses and slower titration suggested in older patients (see label). Cobenfy use is contraindicated in moderate to severe liver impairment and is not recommended in patients with mild liver impairment. It is also not recommended in patients with moderate and severe kidney impairment due to anticholinergic adverse reactions.
• Approval was supported by data from Phase 3 EMERGENT-2 and EMERGENT-3 trials. Cobenfy met the primary study endpoint and demonstrated a significant 9.6-point reduction and 8.4-point reduction in PANSS total score and schizophrenia symptoms compared to placebo at week 5 in EMERGENT-2 and EMERGENT-3, respectively. In EMERGENT-2, Cobenfy also demonstrated a statistically significant improvement in illness from baseline to week 5, as measured by the Clinical Global Impression-Severity (CGI-S) score, a secondary endpoint.
• Use is contraindicated in urinary retention, moderate or severe hepatic (liver) impairment, gastric retention, hypersensitivity to Cobenfy or trospium chloride, untreated narrow-angle glaucoma. Warnings and precautions include urinary retention, liver impairment, biliary disease, gastrointestinal motility, and narrow angle glaucoma, among others.
• The most common side effects reported with Cobenfy (at least 5%) were nausea, dyspepsia / GERD (heartburn, acid reflux), constipation, vomiting, hypertension (high blood pressure), abdominal (stomach area) pain, diarrhea, tachycardia (fast heart rate), and dizziness.

FDA Approves Ebglyss for the Treatment of Atopic Dermatitis

This past month the FDA approved Eli Lilly’s Ebglyss (lebrikizumab-lbkz) injection, an interleukin-13 antagonist for the treatment of moderate-to-severe atopic dermatitis (eczema) in adults and children 12 years and older who weigh at least 40 kg (88 Ibs). It is used in patients that are not well-controlled with topical prescription therapies or when they aren’t advised.

• In atopic dermatitis, a protein called interleukin 13 (IL-13) is involved in the skin inflammation. Ebglyss works by blocking IL-13 to reduce inflammation and improve atopic dermatitis symptoms like itching, redness, irritation and dry skin.
• Ebglyss is given as a subcutaneous injection of two 250 mg injections (500 mg total) at Week 0 and Week 2, followed by 250 mg every 2 weeks until Week 16 or later, based on clinical response. The maintenance dose is 250 mg subcutaneous every 4 weeks. Ebglyss can be used with or without topical corticosteroids.
• Ebglyss comes as a 250 mg / 2 mL single-dose prefilled pen or prefilled syringe. Patients or caregivers may self-inject after training in the subcutaneous injection technique
• FDA approval of Ebglyss was based on results from ADvocate 1, ADvocate 2, and ADhere studies. Two studies (ADvocate 1 and 2) showed that 38% of people who took Ebglyss achieved clear or almost-clear skin at 16 weeks (versus 12% with placebo), and 10% saw these results as early as 4 weeks.
• Warnings and precautions include severe allergic reactions, serious eye problems (conjunctivitis and keratitis), parasitic infections and avoiding use of live vaccines during treatment.
• The most common Ebglyss side effects are eye and eyelid inflammation including redness, swelling, and itching (conjunctivitis), injection site reactions and shingles (herpes zoster).

FDA Approves Boruzu, a Ready-to-Use Bortezomib Injection for Multiple Myeloma and Mantle Cell Lymphoma

In September, Amneal Pharmaceuticals and Shilpa announced approval of Boruzu (bortezomib), the first ready-to-use bortezomib injection to treat the blood cancers multiple myeloma (MM) or mantle cell lymphoma (MCL) in adults.

• Boruzu belongs to a class of medicines known as proteasome inhibitors. It works by blocking the effects of proteasome enzymes which allows damaged proteins to build up and kill the cancer cell. It is considered a targeted drug treatment.
• Boruzu is a ready-to-use formulation and will reduce the steps needed to prepare Velcade, a lyophilized powder form of bortezomib requiring reconstitution before use.
• Boruzu doses are based on weight and given as either a subcutaneous injection (under the skin) or intravenous injection (into a vein) once or twice weekly, based on use and cycle. It is supplied as a single-dose vial (3.5 mg / 1.4 mL [2.5 mg/mL]).
• Healthcare providers should use caution when calculating the volume to be administered because each route of administration has a different final concentration. Use a lower starting dose for patients with moderate or severe hepatic impairment.
• Warnings and precautions include: peripheral neuropathy, low blood pressure, cardiac (heart) toxicity, and lung toxicity, among others.
• The most commonly reported adverse reactions (in at least 20% of patients) in clinical studies include nausea, diarrhea, thrombocytopenia (low platelets), fatigue, low red or white blood cells, constipation, vomiting, rash, pyrexia (fever), and anorexia (appetite loss).
• Boruzu is expected to be available commercially in the second quarter of 2025.

FDA Approves FluMist as First Flu Vaccine That Can Be Administered at Home

The FDA has approved FluMist (Influenza Vaccine Live, Intranasal) as the first flu vaccine for self or caregiver administration. A prescription is still required to receive FluMist, which can be accessed through a third-party online pharmacy. FluMist is approved for the prevention of influenza (“flu”) disease caused by influenza virus subtypes A and B in individuals 2 through 49 years of age.

• Influenza (the “flu”) is a common and contagious fall and winter viral respiratory illness in the U.S. Symptoms include body aches, fatigue, fever, sore throat, stuffy nose or coughing. In older patients, those with certain chronic conditions, or young children the flu can be severe and life-threatening. The viral illness caused up to 51,000 deaths between 2010 and 2023, according to the CDC.
• FluMist is sprayed into the nose and was initially approved by the FDA in 2003 for use in individuals 5 through 49 years of age. In 2007 the FDA approved the use of FluMist to include children 2 through 5 years of age.
• ​​FluMist may now be administered by a health care provider in a health care setting (including a pharmacy) OR it may be administered by the vaccine recipient or a caregiver who is 18 years of age or older.
• MedImmune, the manufacturer, plans to make the vaccine available through a third-party online pharmacy.
• After eligibility screening, the pharmacist will approve the prescription and ship the vaccine to the recepient, along with labeling information (prescribing information, information for patients / caregivers and instructions for use). Only a caregiver should administer FluMist to individuals 2 through 17 years of age.
• Home delivery of FluMist is anticipated to be available next year for the 2025-2026 flu season.
• The most commonly reported side effects of FluMist are fever over 100°F in children 2 through 6 years of age, runny nose and nasal congestion in individuals 2 through 49 years of age and a sore throat in adults 18 through 49 years of age.

Posted September 2024

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