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FDA Updates for the Week of Sept. 18, 2023


The FDA has approved GSK’s myelofibrosis drug, as well as a new indication for Jardiance and a new dosing regimen for Talicia. The agency has also issued a complete response letter for epinephrine nasal spray and assigned a review date for the gene therapy atidarsagene autotemcel.

FDA Approves GSK’s Myelofibrosis Drug

The FDA has approved GSK’s Ojjaara (momelotinib) to treat adult patients with anemia who have intermediate or high-risk myelofibrosis. Myelofibrosis is a cancer of the bone marrow and can lead to anemia. It affects about 25,000 patients in the United States, and about 40% of patients have moderate to severe anemia at the time of diagnosis. These patients often require transfusions, and those who do have a poor prognosis and shortened survival.

Ojjaara has the ability to inhibit three key signaling pathways: Janus kinase (JAK) 1, and JAK2 and activin A receptor, type I (ACVR1). Inhibition of JAK1 and JAK2 may improve symptoms and enlarged spleen, and direct inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anemia, according to GSK.

Ojjaara will be available soon, according to a GSK spokesperson. The wholesale acquisition cost is $26,900 for a bottle of 30 tablets. The spokesperson said the price of Ojjaara reflects the value it will deliver to patients, healthcare systems, and society and takes into consideration the fact it is the only medicine that addresses key manifestations of myelofibrosis, including anemia, constitutional symptoms, and splenomegaly. GSK offer access and reimbursement services for all GSK oncology products at TogetherWithGSKOncology.com. For Ojjaara, GSK offers $0 copay assistance, patient assistance for uninsured patients and a bridge program for those experiencing delays in insurance coverage.

FDA Approves New Dosing Regimen for the H. Pylori Drug Talicia

The FDA has approved a new dosing option for RedHill Biopharma’s Talicia to treat patients with Helicobactor pylori infection. The new regimen is three times daily, taken at least four hours apart with food. This allows for a “breakfast, lunch and dinner” routine, which the company said helps to support increased patient adherence. The previous dosing was every eight hours with food.

H. pylori is a bacterial infection of the stomach that affects about 35% of the U.S. population. Most people don’t have any symptoms from H. pylori infection, but for those who do, symptoms include stomach pain, nausea, loss of appetite, bloating and unintentional weight loss. H. pylori can lead to stomach ulcers and is a risk factor for certain types of stomach cancer. The World Health Organization classifies H. pylori as a group 1 carcinogen.

Talicia a fixed-dose, all-in-one oral capsule combination of two antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor (omeprazole). It is the only FDA-approved rifabutin-based therapy for the eradication of H. pylori, and company officials said it was designed to address H. pylori resistance to clarithromycin. It has retail cost of $816 for a supply of 168 capsules, according to Drugs.com.

FDA Approves Jardiance for Patients with Chronic Kidney Disease

The FDA has approved Jardiance (empagliflozin) to treat adults with chronic kidney disease. Jardiance is currently available to treat patients with type 2 diabetes, end stage renal disease and heart failure. Developed by Boehringer Ingelheim and Lilly, Jardiance has a list price of $570.48 for a month supply.

Chronic kidney disease affects more than one in seven adults in the United States, 90% of whom are undiagnosed, and it remains a significantly under-recognized public health crisis.

FDA Issues Complete Response for Epinephrine Nasal Spray

The FDA has issued a complete response letter (CRL) for ARS Pharmaceuticals’ new drug application (NDA) for neffy (epinephrine nasal spray) to treat patients with allergic reactions that could lead to anaphylaxis. ARS officials said in a press release they plan to appeal.

The FDA has requested a pharmacokinetic/pharmacodynamic (PK/PD) study assessing repeat doses of neffy compared with repeat doses of an epinephrine injection under allergic rhinitis conditions. PK/PD studies provide information about the drug within the body. Pharmacokinetics assesses what the body does to the drug (adsorption, distribution, metabolism and excretion), and pharmacodynamics assess what the drug does to the body (how the body responds).

This request comes after the recommendation of an FDA advisory committee in May 2023 to approve neffy without the need for additional studies to demonstrate its efficacy or safety. Previously, the FDA and ARS Pharma had agreed on the labeling and a postmarketing requirement to conduct this study.

FDA Assigns Review Date for Gene Therapy Atidarsagene Autotemcel

The FDA has accepted for priority review the biologics license application (BLA) for atidarsagene autotemcel, also known as OTL-200, to treat patients with metachromatic leukodystrophy (MLD). The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

Metachromatic leukodystrophy is a rare and life-threatening inherited disease of the body’s metabolic system. It is caused by a mutation in the arylsulfatase-A (ARSA) gene, which results in the accumulation of sulfatides in the brain and other areas of the body. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see.

Developed by Orchard Therapeutics, atidarsagene autotemcel is one-time gene therapy. Patients stem cells are removed and are genetically modified. They are then returned to the patient by intravenous infusion to deliver the corrected version of the gene. Orchard is seeking approval for early onset metachromatic leukodystrophy, which has a five-mortality rate of 50%. The FDA had previously given OTL-200 both Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations.

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