FDA Updates for the Week of Sept. 26, 2022

FDA approves Amylyx’s ALS therapy.

The FDA has approved Amylyx Pharmaceuticals’ AMX0035 — now with the brand name Relyvrio (sodium phenylbutyrate/taurursodiol) — to treat patients with amyotrophic lateral sclerosis (ALS). It is an oral, fixed-dose combination therapy that should be available within four to six weeks, the company said in a call with investors. It will be available through specialty pharmacies, and can be used as a monotherapy or with other therapies.

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. It is a progressive disease, and most cases will result in death from respiratory failure, usually within three to five years from when the symptoms first appear. About 5,000 people in the United States are diagnosed with ALS annually and about 29,000 people are currently living with ALS.

The company formed the Amylyx Care Team, which will provide a single point of contact for patients and help with financial assistance. The wholesale acquisition cost will be $158,000 per year for the first year, which company officials said is below the last approved ALS therapy. Mitsubishi Tanabe Pharma’s Radicava (edaravone), which was approved in 2017, costs about $171,000 a year.

According to an analysis from the Institute for Clinical and Economic Review, the therapy would only achieve traditional thresholds of cost-effectiveness if priced between $9,100 to $30,700 per year.

FDA approves fourth Avastin biosimilar.

The FDA has approved Celltrion Healthcare’s Vegzelma (bevacizumab-adcd), a biosimilar to Genentech’s Avastin (bevacizumab), for the treatment of six types of cancer: metastatic colorectal cancer; recurrent or metastatic non-squamous non-small cell lung cancer (NSCLC); recurrent glioblastoma; metastatic renal cell carcinoma; persistent, recurrent, or metastatic cervical cancer; and epithelial ovarian, fallopian tube, or primary peritoneal cancer.

The FDA approval of Vegzelma was based on the totality of evidence, including the pivotal phase III trial in patients with metastatic or recurrent non-squamous NSCLC. Results showed that as a first-line treatment, Vegzelma is highly similar to the reference product in terms of efficacy, safety and pharmacokinetics.

Vegzelma is Celltrion’s third oncology biosimilar approved for use in the United States following the approval of Truxima (rituximab-abbs) and Herzuma (trastuzumab-pkrb). Vegzelma was approved in the EU in August 2022 and UK and Japan in September 2022.

FDA approves Duxipent for rare skin disorder.

The FDA has approved Dupixent (dupilumab) injection for the treatment of adults with prurigo nodularis (PN). This is the first FDA-approved treatment for prurigo nodularis, a skin disease that causes hard, itchy nodules to form on the skin. The itching can be intense, causing people to scratch themselves to the point of bleeding or pain. The disease affects about 87,000 adults per year according to the National Organization for Rare Diseases database.

This approval represents the second dermatology indication for Dupixent and fifth disease indication overall in the United States. Developed by Sanofi and Regeneron Pharmaceuticals, Dupixent is a human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. It is approved for multiple indications, including treatment of atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, and eosinophilic esophagitis. Dupixent’s most recent approval was in June 2022 for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis.

FDA approves Omlonti for glaucoma

The FDA has approved Omlonti (omidenepag isopropyl) eye drops for the reduction of elevated intraocular pressure (IOP) in patients with primary open-angle glaucoma or ocular hypertension. Omlonti, developed jointly by Japanese-based Santen Pharmaceutical and UBE, is a relatively selective prostaglandin EP2 receptor agonist.

Glaucoma causes damage to the optic nerve and remains a leading cause of irreversible blindness worldwide; it is estimated to impact about 80 million people worldwide. Primary open angle glaucoma is the predominant subtype of glaucoma. Increased intraocular pressures is an important risk factor for glaucoma.

FDA accepts NDA for therapy for rare immunodeficiency disease.

The FDA has accepted for priority review Pharming’s new drug application (NDA) for leniolisib to treat patients 12 years and older with a rare primary immunodeficiency disease. The disease, activated phosphoinositide 3-kinase delta syndrome (APDS), affects 1 to 2 people per million. It is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023.

Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. Leniolisib inhibits the production of PIP3, which is an important cellular messenger and it regulates cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism.

The NDA was supported by positive data from a phase 2/3 study of leniolisib, which met its co-primary endpoints of reduction in index lymph node size and correction of immunodeficiency in the target population. The findings indicate a reduction in disease markers associated with APDS, which include significant lymphoproliferation and immune dysfunction, as well as increased risk of lymphoma.