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FDA Updates for the Week of June 19, 2023


The FDA’s approvals this week include: a $3.2 million gene therapy, the first anti-inflammatory drug for cardiovascular disease, a subcutaneous version of Vyvgart for myasthenia gravis and Jardiance and Synjardy for kids with type 2 diabetes. Three rejections by the agency include: a therapy for NASH, for retinal cancer, and for rare fungal infections. In addition, Geron has submitted an NDA for first-in-class therapy for MDS.

First Gene Therapy for Duchenne Priced at $3.2 Million.

The first FDA-approved gene therapy to treat Duchenne muscular dystrophy (DMD). Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), will have a wholesale acquisition cost (WAC) of $3.2 million. The FDA granted accelerated approval of Elevidys to treat ambulatory pediatric patients aged 4 through 5 years with a confirmed mutation in the DMD gene. This follows a positive vote by an advisory committee in May.

Elevidys is a one-time therapy that delivers to muscle a gene that codes for a shortened, functional form of dystrophin. It uses an adeno-associated virus (AAV) vector and a promoter that helps to drive gene expression.

The FDA is requiring the company to complete a clinical study to confirm the drug’s clinical benefit. Sarepta is conducting a placebo-controlled phase 3 trial for Elevidys will serve as the confirmatory trial and is fully enrolled with top-line results expected in late 2023.

FDA Approves First Anti-inflammatory Drug for Cardiovascular Disease.

The FDA has approved Lodoco (colchicine) to reduce the risk of myocardial infarction (MI), stroke, coronary revascularization, and cardiovascular death in adult patients. Developed by Agepha Pharma, Lodoco is the first drug approved to target inflammation that is an underlying cause of atherosclerotic cardiovascular disease. It can be used alone or in combination with cholesterol-lowering medications.

Colchicine is a well-established molecule and generic colchicine that is used at high doses for the treatment of gout. Lodoco has been reformulated in a lower dose specifically for long-term use in cardiovascular disease patients. Lodoco 0.5 mg tablets are a once-daily tablet and will be available in the second half of 2023. An exact price hasn’t been determined.

FDA Approves Subcutaneous Vyvgart for Myasthenia Gravis.

The FDA has approved Argenx’s Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) to treat patients with generalized myasthenia gravis. It is indicated for adult patients who are anti-acetylcholine receptor antibody positive, which represents about 85% of the total generalized myasthenia gravis population. Generalized myasthenia gravis is a rare autoimmune disease that leads to weakness in skeletal muscles, causing muscles to tire and weaken.

Vyvgart intravenous was approved in December 2021, and it binds to the neonatal Fc receptor (FcRn) to reduce circulating IgG. Vyvgart Hytrulo, a subcutaneous (under the skin) therapy, adds the recombinant human hyaluronidase PH20 to efgartigimod alfa. It uses Halozyme’s ENHANZE drug delivery technology to facilitate subcutaneous delivery. The addition of human hyaluronidase PH20 means the therapy can be delivered in about 30 to 90 seconds compared with the intravenous therapy, which takes about an hour. Vyvgart Hytrulo is the first approved FcRn blocker administered by subcutaneous injection. It is expected to be available in July 2023.

The typical net price for Vyvgart is about $225,000 annually, and Argenx indicates Vyvgart Hytrulo will be priced at parity. The company provides copay assistance with a maximum of $25,000 a year.

FDA Clears Jardiance and Synjardy for Kids with Type 2 Diabetes.

The FDA has approved Boehringer Ingelheim and Lilly’s Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin) to improve blood sugar control in children 10 years and older with type 2 diabetes, marking a new class of medicines taken by mouth to treat pediatric type 2 diabetes.

The blockbuster diabetes medication Jardiance was also granted an additional heart failure indication in February 20226. The current list price for a month supply of Jardiance is $570.48.

FDA Rejects NDA for Fatty Liver Disease Therapy.

The FDA has issued a complete response letter for the new drug application for obeticholic acid to treat patients with pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH). NASH is a progressive liver disease caused by excessive fat accumulation in the liver that leads to inflammation and liver injury. Developed by Intercept Pharmaceuticals, obeticholic acid is a farnesoid X receptor (FXR) agonist.

Last month, an FDA advisory committee voted no on the question of whether the efficacy and safety presented outweigh the risks. In its CRL, the FDA indicated that any resubmission of an NDA for OCA in NASH would require data on long-term outcomes.

The company has decided to discontinue all NASH-related research and restructured to focus on rare and serious liver diseases. R&D will now focus on a fixed-dose combination of OCA and bezafibrate to treat patients with primary biliary cholangitis (PBC), a chronic disease that affects the bile ducts in the liver.

FDA Issues CRL for Treatment for Rare Retinal Cancer.

The FDA has issued a complete response letter to Aldeyra Therapeutics on its new drug application of ADX-2191 (methotrexate) to treat patients with primary vitreoretinal lymphoma (PVRL). The agency indicated there is a lack of evidence of effectiveness and lack of clinical trials done to support the application. Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that is diagnosed in about 300 to 600 patients in the United States per year.

Aldeyra did not conduct clinical trials specifically in patients with primary vitreoretinal lymphoma. Its NDA was based on a combination of published literature on the safety and efficacy of methotrexate in patients with primary vitreoretinal lymphoma, as well as safety data from the completed phase 3 GUARD Trial in patients with proliferative vitreoretinopathy, a complication of retinal detachment. Aldeyra plans to discuss ADX-2191 for the treatment of PVRL with the FDA, including the potential to make it accessible these patients under an Expanded Access Program protocol.

FDA Issues Complete Response for Drug for Rare Fungal Infections.

The FDA has issued a complete response letter for F2G’s new drug application (NDA) for olorofim to treat patients with invasive fungal infections. The agency is requesting additional data and analyses.

Olorofim is being developed for difficult-to-treat invasive, rare fungal mold infections such as azole-resistant aspergillosis, scedosporiosis and lomentosporiosis. Invasive fungal infections can cause morbidity and mortality, particularly among immunosuppressed patients. Effective therapies are limited, and even when therapies exist, some patients may become resistant. Olorofim works through a novel mechanism of action, different from existing classes of antifungals, by inhibiting the enzyme dihydroorotate dehydrogenase in the pyrimidine synthesis pathway, a pathway for DNA synthesis.

Geron Submits NDA for First-in-Class Therapy for MDS.

Geron has submitted a new drug application to the FDA for imetelstat to treat adult patients with transfusion-dependent anemia. If approved, it would indicated for patients with low- to intermediate-risk for myelodysplastic syndromes (MDS) who have failed to respond to erythropoiesis-stimulating agents. Myelodysplastic syndromes are a group of cancers in which blood stem cells do not mature. This can lead to anemia, infection or bleeding.

Imetelstat is a first-in-class therapy that targets telomerase to inhibit uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies.

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