LENMELDY™

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…

TEVIMBRA

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.


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Poor Trial Results May Prompt Maker to Pull ALS Drug Relyvrio From Market

Courtesy of HealthDay, by Robin Foster, HealthDay Reporter; Medically reviewed by Carmen Pope, BPharm

Following disappointing trial results, the maker of a controversial ALS drug may pull the medication off the market.

In a statement issued Friday, Amylyx Pharmaceuticals said that Relyvrio failed to help patients in a large follow-up study, but the company stopped just short of saying it will definitely pull the drug from the market. The drug is sold as Albrioza in Canada.

“Amylyx intends to share plans for Relyvrio/Albrioza in ALS, which may include voluntarily withdrawing Relyvrio/Albrioza from the market,” the company said in its statement. “At this time, Relyvrio/Albrioza and its related patient support program will continue to be available for people living with ALS. Amylyx has voluntarily decided to pause promotion of the medication during this time.”

Executives added that they were “surprised and disappointed” by the results and would announce their plans for the drug in the next two months.

Relyvrio was first approved by the U.S. Food and Drug Administration (FDA) in September 2022, following a lengthy, impassioned campaign by patients with ALS, a fatal muscle-wasting disease.

Unfortunately, the latest company study showed the drug did not slow the disease compared with a placebo treatment, and it also did not produce improvements on any secondary measures, such as muscle strength.

ALS is a devastating neurological disease that slowly destroys nerve cells and connections that are needed to walk, talk, speak, and breathe. Most patients die within three to five years of their diagnosis.

Relyvrio’s approval was mainly based on results from one small study that was criticized by some of the FDA’s scientists. An outside committee of experts also voted against the drug initially, before being swayed to back it at a follow-up meeting requested by patients. At the time, Amylyx noted it was continuing a larger follow-up study of more than 600 patients that would provide further data on the drug.

In a highly unusual move, Amylyx executives at that second advisory committee meeting told FDA regulators they would pull the drug from the market if follow up research didn’t confirm its efficacy. That commitment seemed to reassure FDA’s advisers, who then voted in favor of the drug’s approval, despite the questionable data.

Amylyx has also faced criticism for pricing the drug at $158,000 for a year’s supply, the Associated Press reported, and sales have been lackluster since Relyvrio’s launch in late 2022. Relyvrio combines two older drugs: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine.

Sources
• Amylyx Pharmaceuticals, news release, March 8, 2024
• Associated Press

TYENNE

FDA Approves Tyenne (tocilizumab-aazg), a Biosimilar to Actemra

Fresenius Kabi, announced today that the U.S. Food and Drug Administration (FDA) has approved Tyenne® (tocilizumab-aazg), its tocilizumab biosimilar referencing Actemra® (tocilizumab). Tyenne becomes the first tocilizumab biosimilar with both IV and subcutaneous formulations approved by the FDA. In accordance with its patent settlement agreement with Genentech, Fresenius Kabi has a license to market its tocilizumab products in the U.S. commencing on the license dates, which are confidential.

Tyenne has launched globally in more than 10 countries, with plans to launch in many more countries between 2024 and 2025. Tyenne (tocilizumab-aazg) is FDA approved for the treatment of several inflammatory and immune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis.

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JUBBONTI

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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WYOST

FDA Approves Wyost (denosumab-bddz), an Interchangeable Biosimilar to Xgeva

Sandoz, announced that the U.S. Food and Drug Administration (FDA) approved Wyost (denosumab-bbdz), an interchangeable biosimilar to Xgeva. Wyost is approved to prevent skeletal-related events (SREs) in patients with multiple myeloma and in patients with bone metastases from solid tumors, to treat adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity, and to treat hypercalcemia of malignancy refractory to bisphosphonate therapy.

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DEFINITY®

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®

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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LETYBO

FDA Approves Letybo (letibotulinumtoxinA-wlbg) for the Treatment of Glabellar Lines

Hugel America, Inc., a division of Hugel Inc., today announced it received U.S. Food and Drug Administration (FDA) approval on its neurotoxin, Letybo to treat moderate-to-severe glabellar (frown) lines in adults. In the coming months, Hugel plans to accelerate its transition for approved medical use with the goal of launching to aesthetic clinicians in the back half of 2024.

FDA approval is supported by positive results from three completed phase III trials that enrolled more than 1,000 subjects in the U.S. and Europe. This botulinum toxin type A has been the leading neurotoxin brand in South Korea, one of the world’s most dynamic aesthetic markets, for seven consecutive years. Hugel has sold over 26 million vials in 50+ different countries including Australia, Canada, and Europe.

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