Posts in category: Uncategorized

FDA Grants Accelerated Approval for Rezdiffra for the Treatment of Patients with Noncirrhotic Nonalcoholic Steatohepatitis (NASH)

Madrigal Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Rezdiffra (resmetirom) in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH), with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). Continued approval for this indication may be contingent upon verification and description of clinical benefits in ongoing confirmatory trials.

Rezdiffra is a once-daily, oral THR-beta agonist designed to target key underlying causes of NASH. The accelerated approval of Rezdiffra was based on results from the Phase 3 MAESTRO-NASH trial, which was recently published in the New England Journal of Medicine.

The Rezdiffra prescribing information does not include a liver biopsy requirement for diagnosis. Rezdiffra should not be used in patients with decompensated cirrhosis.

Read more at Drugs.com…

FDA Approves Lenmeldy for Children with Metachromatic Leukodystrophy

Orchard Therapeutics/Kyowa Kirin has announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™, formerly known as OTL-200, for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Metachromatic leukodystrophy is a rare and severe genetic condition that affects the brain and nervous system. Metachromatic leukodystrophy is caused by a faulty gene, which means that the body does not make enough of an enzyme called arylsulfatase A (ARSA). That lack of ARSA causes fatty substances called sulfatides to build up in the brain and nerves, leading to problems with movement and thinking abilities, severe spasticity, seizures, and patients gradually losing the ability to move, talk, swallow, eat, and see.

Lenmeldy is a gene therapy used to treat of types of metachromatic leukodystrophy in children. Lenmeldy is made using the patient’s own stem cells that have been modified so that the body can make the ARSA enzyme to help stop the progression of MLD. Lenmeldy is given as a one-time infusion.

Read more at Drugs.com…

FDA Approves Tevimbra for the Treatment of Advanced or Metastatic Esophageal
Squamous Cell Carcinoma

BeiGene, Ltd., has announced that the U.S. Food and Drug Administration (FDA) has approved Tevimbra (tislelizumab-jsgr) as monotherapy for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.

Approval is based on the RATIONALE 302 trial, which met its primary endpoint in the intention-to-treat (ITT) population with a statistically significant and clinically meaningful survival benefit for Tevimbra compared with chemotherapy, highlighting its potential as an important treatment option for these patients.

Tevimbra is an IV injection and will be available in the U.S. in the second half of 2024.

Read more at Drugs.com…

Instead of approving the new Alzheimer’s drug donanemab this month, as was expected, the U.S. Food and Drug Administration (FDA) will now require the experimental medication be scrutinized more closely by an expert panel.

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FDA Approves Jubbonti (denosumab-bddz), an Interchangeable Biosimilar to Prolia

Sandoz, has announced that the U.S. Food and Drug Administration approved Jubbonti, (denosumab-bbdz), an interchangeable biosimilar to Prolia.

Jubbonti is approved to:

• Treat postmenopausal women with osteoporosis at high risk for fracture.

• Increase bone mass in men with osteoporosis at high risk for fracture.

• Treat glucocorticoid-induced osteoporosis in men and women at high risk for fracture.

• Increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer.

• Increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer.

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Lantheus Announces the FDA Approval of Definity (Perflutren Lipid Microsphere) for Pediatric Patients
Lantheus Holdings, Inc, has announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application (sNDA) for Definity® (Perflutren Lipid Microsphere) as an ultrasound enhancing agent for use in pediatric patients with suboptimal echocardiograms. This approval represents a significant step forward in pediatric medicine, providing healthcare professionals with a valuable tool to opacify the left ventricular chamber and better identify the left ventricular endocardial border. Currently, Definity is the most utilized, extensively studied, and a trusted diagnostic ultrasound enhancing agent in the U.S.

Clinical studies have substantiated the efficacy and safety of Definity in pediatric patients.

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Rybrevant® in Combination with Chemotherapy Is the First FDA Approved Therapy for First-line Treatment of Patients With Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

Johnson & Johnson has announced that following a priority review, the U.S. Food and Drug Administration (FDA) has approved Rybrevant® (amivantamab-vmjw) in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with locally advanced or metastatic nonsmall cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test. This FDA action converts the May 2021 accelerated approval of Rybrevant® to a full approval based on the confirmatory Phase 3 PAPILLON study.

Worldwide, lung cancer is one of the most common cancers, with NSCLC making up 80 to 85 percent of all lung cancer cases. Alterations in EGFR are the most common actionable driver mutations in NSCLC. Clinical data show patients with EGFR exon 20 insertion mutations generally experience limited benefits with currently approved third-generation EGFR tyrosine kinase inhibitors and chemotherapy. NSCLC driven by EGFR exon 20 insertion mutations carries a worse prognosis and shorter survival rates compared with lung cancer driven by other EGFR driver mutations.

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U.S. FDA Approves Expanded Indication for Gilead’s Biktarvy to Treat People with HIV with Suppressed Viral Loads, Pre-Existing Resistance

Gilead Sciences, Inc. has announced the U.S. Food and Drug Administration (FDA) approved a new, expanded indication for Biktarvy® (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg tablets, B/F/TAF) to treat people with HIV (PWH) who have suppressed viral loads with known or suspected M184V/I resistance, a common form of treatment resistance. HIV treatment resistance is permanent and irreversible, which can jeopardize future treatment options for PWH. The M184V/I resistance mutation has been found to be present in a range (22-63%) of PWH with pre-existing resistance to nucleoside reverse transcriptase inhibitors (NRTIs) across various HIV subtypes.

This label update is supported by Study 4030, which evaluated the efficacy, safety, and tolerability profile of Biktarvy in a broad range of people with HIV-1 with or without pre-existing NRTI resistance, including those with the M184V/I resistance. Biktarvy is now the first and only integrase strand transfer inhibitor (INSTI)-based single-tablet regimen that is FDA approved and U.S. Department of Health and Human Services (DHHS) guideline recommended for PWH who are virally suppressed with M184V/I resistance.

Once someone with HIV has developed resistance to a treatment, it will persist for the rest of their life. Reducing the risk of drug resistance is a key goal in HIV therapy. HIV drug resistance continues to receive clinical and public health attention because it may hinder the ability of HIV medicines to suppress and block replication of the virus over the course of an individual’s life. Resistance may lead to treatment failure in individuals, while also creating the potential for transmission of treatment-resistant HIV within communities.

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FDA Approves Simlandi (adalimumab-ryvk), an Interchangeable Biosimilar to Humira

Alvotech and Teva Pharmaceuticals, have announced that the U.S. Food and Drug Administration (FDA) has approved Simlandi (adalimumab-ryvk) injection, as an interchangeable biosimilar to Humira, for the treatment of adult rheumatoid arthritis, juvenile idiopathic arthritis, adult psoriatic arthritis, adult ankylosing spondylitis, Crohn’s disease, adult ulcerative colitis, adult plaque psoriasis, adult hidradenitis suppurativa and adult uveitis. In 2023, Humira was one of the highest-grossing pharmaceutical products in the world, with sales in the U.S. of nearly $12.2 billion. Teva is Alvotech’s strategic partner for the exclusive commercialization of Simlandi in the United States.

Simlandi is the first high-concentration, citrate-free biosimilar to Humira that has been granted an interchangeability status by the FDA and will qualify for interchangeable exclusivity for the 40mg/0.4ml injection. While both low-concentration and high-concentration strength biosimilars of Humira are marketed in the U.S. today, nearly 88 percent of U.S. prescriptions for adalimumab are for the highconcentration presentation.

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FDA Approves new indication for Xolair

The U.S. Food and Drug Administration (FDA) approved a new indication for Genentech/Novartis’ Xolair (omalizumab), an anti-immunoglobulin E (IgE) antibody. Xolair is now indicated for IgE-mediated food allergy in adult and pediatric patients 1 year of age and older for the reduction of allergic reactions (Type I). This includes anaphylaxis, that may occur with accidental exposure to one or more foods. Xolair is intended to be used in conjunction with food allergen avoidance.

Xolair was first approved in 2003 for moderate to severe persistent allergic asthma and has since been approved for perennial aeroallergen allergies, to treat chronic idiopathic urticaria, and as an add-on maintenance treatment for nasal polyps in adults with inadequate response to nasal corticosteroids. Xolair does not treat acute allergic food reactions and does not replace the use of epinephrine for such reactions. Patients taking Xolair for food allergies may need to continue to carry epinephrine for treating emergency allergic reactions.

Xolair is the first FDA-approved medication to reduce allergic reactions to more than one type of food after accidental exposure. Aimmune Therapeutics’ Palforzia (peanut [Arachis hypogaea] allergen powder-dnfp) is an oral immunotherapy product approved in patients 4–17 years of age for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut, but its use is restricted to peanut allergy. There are other agents in development for food allergies, including some for multiple food allergies.

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