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FDA Updates for Week of March 18, 2024: A $4.25 Million Gene Therapy and Drugs for Duchenne and Leukemia


New approvals this week include a $4.25 million gene therapy, a drug that treats hypertension in a new way, a nonsteroidal drug for Duchenne, and an oral drug for aggressive leukemia. The agency has set an action date for resubmitted Lymphir in rare skin cancer, and Celltrion has launched Zymfentra, a subcutaneous form of infliximab.

New Gene Therapy for Rare MDL to Cost $4.25 Million

Orchard Therapeutics has set the wholesale acquisition cost of its newly approved gene therapy, Lenmeldy (atidarsagene autotemcel), at $4.25 million for the one-time treatment. Company officials in a press release said this price reflects its clinical, economic and societal value. Lenmeldy is the first approved gene therapy to treat children with juvenile metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system.

Orchard Therapeutics has a patient support program for patients and their caregivers who will assist with both private and public insurance to access Lenmeldy. Infants with MLD qualify for government assistance so state Medicaid programs are expected to be part of the insurance mix. Five treatment centers with specialized expertise in transplant and the treatment of neurometabolic diseases are being activated to administer Lenmeldy.

FDA Approves Drug that Treats Hypertension in a New Way

The FDA has approved Tryvio (aprocitentan) to treat adults with uncontrolled hypertension. It is indicated to be used in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs.

Developed by Idorsia, Tryvio represents a new way to treat hypertension. It is an endothelin receptor antagonist that targets endothelin-1 A/B receptors, which is a major driver of aldosterone production. Current treatments for high blood pressure regulate salt and water (diuretics), reduce extracellular calcium (calcium channel blockers), target the renin–angiotensin–aldosterone (RAAS) system or sympatholytic activity (beta blockers, central alpha-agonist agents), or have non-selective vasodilatory effects.

The recommended dosage of Tryvio is 12.5 mg orally once daily, with or without food. It will be available in the second half of 2024. The list price has not yet to be determined, but the company plans to announce the price in the coming weeks.

FDA Approves Nonsteroidal Drug for Duchenne

The FDA has approved Duvyzat (givinostat) to treat patients with Duchenne muscular dystrophy (DMD) who are six years of age and older.

Duvyzat is the first nonsteroidal oral drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and loss of muscle. Duvyzat was developed by Milan-based Italfarmaco in partnership with Telethon and Duchenne Parent Project (Italy). It will be marketed in the United States by ITF Therapeutics, a new subsidiary of Italfarmaco.

Company officials said they anticipate Duvyzat will be available in the third quarter of 2024. Dosing will be based on weight. The pricing has not yet been determined.

FDA Approves Iclusig for New Patients with Aggressive Leukemia

The FDA has granted an accelerated approval for a supplemental indication for Iclusig (ponatinib). It is now approved to treat adult patients who have been newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL). Iclusig is indicated to be used in combination with chemotherapy. Philadelphia-positive ALL is a type of fast-growing blood cancer that starts in young white blood cells called lymphoblasts or blast cells.

Developed by Takeda, Iclusig is an oral kinase inhibitor that targets BCR-ABL1, an abnormal tyrosine kinase. Iclusig is already approved to treat patients who in are the chronic phase of chronic myelogenous leukemia and the blast phase of Philadelphia chromosome-positive acute lymphoblastic leukemia.

Iclusig has a wholesale acquisition cost of $20,831 for 30 tablets.

FDA Sets Action Date for Resubmitted Lymphir in Rare Skin Cancer

The FDA has accepted the resubmission of the Citius Pharmaceuticals’ biologics license application (BLA) for Lymphir (denileukin diftitox), to treat patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. The FDA has assigned a Prescription Drug User Fee Act goal date of Aug.13, 2024.

Cutaneous T-cell lymphoma is a type of cutaneous non-Hodgkin lymphoma (NHL). In cutaneous T-cell lymphoma, T-cells become cancerous and presents as red, scaly patches on the skin.

Lymphir (previously called I/Ontak) is a recombinant fusion protein that combines interleukin-2 (IL-2) receptors with diphtheria toxin fragments. It binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis.

Celltrion Launches Zymfentra, a Subcutaneous Form of Infliximab

Celltrion USA has launched Zymfentra with a price of $6,181.08 for two shots over four weeks. The FDA had approved Zymfentra (infliximab-dyyb) in October 2023 as the first subcutaneous formulation of infliximab to be used a maintenance therapy for adults with moderate-to-severe active ulcerative colitis and Crohn’s disease.

Celltrion provides commercially insured patients with copay assistance of $5 per month for eligible patients. This is subject to a maximum annual limit, but so far, this limit is not listed on the Zymfentra product site.

Although Inflectra is an infliximab biosimilar that references Remicade, Zymfentra was approved through the stand-alone biologics license application process. It is considered a new drug due to the subcutaneous administration. It has patent protection through 2037 for its dosage form and 2040 for its route of administration.

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