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FDA Updates for the Week of Feb. 12, 2024: New Approvals, an Extended Review and Goal Dates Set


The FDA approves Eohilia for eosinophilic esophagitis and Onivyde for metastatic pancreatic cancer but extends review of gene therapy for rare immune disorder. The agency also set goal dates for three products: full approval for Elevidys, Augtyro for solid tumors with NTRK gene fusions and seladelpar as for rare liver disease.

FDA Approves Eohilia as an Oral Treatment for Eosinophilic Esophagitis

The FDA has approved Takeda’s Eohilia (budesonide), an oral therapy for people 11 years and older with eosinophilic esophagitis (EoE). It will be available in 2 mg/10 mL single-dose, stick packs by the end of February.

Eosinophilic esophagitis is an inflammatory disease of the esophagus, causing it to narrow and develop abscesses. Although the exact cause is unknown, it is believed to be triggered by a variety of stimuli including certain foods and environmental allergens. The chronic inflammation of EoE can lead to a range of symptoms, which can vary by person and age, and include difficulty swallowing, vomiting and pain.

Eohilia is a corticosteroid indicated for 12 weeks of treatment. It is a liquid formulation of budesonide and will have a wholesale acquisition cost of $1,875 per month.

The FDA approval of Eohilia is based on efficacy and safety data from two multicenter, randomized studies in patients — ages 11 to 56 in study one and 11 to 42 in study two. In both studies, patients received at least one dose of either Eohilia 2 mg or placebo orally twice daily.

FDA Approves Onivyde for First-line Metastatic Pancreatic Cancer

The FDA has approved Ipsen’s supplemental new drug application for Onivyde (irinotecan liposome) as a first-line treatment for adults with metastatic pancreatic adenocarcinoma. It is indicated to be used with the chemotherapy regimen oxaliplatin, fluorouracil and leucovorin (NALIRIFOX)

Pancreatic adenocarcinoma is the most common type of cancer that forms in the pancreas, with more than 60,000 people diagnosed in the United States each year. It is often detected after the disease has spread and has a poor prognosis with fewer than 20% of people surviving longer than one year.

Onivyde blocks an enzyme called topoisomerase I, which is involved in copying cell DNA needed to make new cells. It is administered via intravenous infusion over 90 minutes every two weeks. It is already approved in combination with fluorouracil and leucovorin to treat patients with metastatic pancreatic adenocarcinoma after disease progression following gemcitabine-based therapy.

The current FDA approval was based on efficacy and safety data from NAPOLI 3, a phase 3 pivotal trial that enrolled 770 patients with metastatic pancreatic adenocarcinoma without prior treatment.

FDA Extends Review of Gene Therapy for Rare Immune Disorder

The FDA has extended the priority review period for Rocket Pharmaceuticals’ biologics license application (BLA) for Kresladi (marnetegragene autotemcel or RP-L201) to treat the rare, autosomal recessive pediatric disease leukocyte adhesion deficiency-I (LAD-I). The application is being delayed by three months for regulators to review clarifying chemistry, manufacturing, and controls (CMC) information submitted by Rocket. The new Prescription Drug User Fee Act (PDUFA) date is now June 30, 2024.

Severe leukocyte adhesion deficiency-I is caused by mutations in the ITGB2 gene that encodes for CD18, a key protein that facilitates the immune response against infections. As a result, white blood cells (leukocytes) do not function normally. Children with this disease experience life-threatening bacterial and fungal infections that respond poorly to antibiotics. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. LAD-I is estimated to impact an estimated 800 to 1,000 children in the United States and Europe.

Kresladi is a lentiviral vector-based investigational gene therapy.

FDA Sets Date for Full Approval, Broader Indication for Elevidys

The FDA has granted priority review for Sarepta Therapeutics' supplemental biologics license application for the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) and set a review goal date of June 21, 2024. Elevidys was granted an accelerated approval in June 2023 to treat ambulatory patients aged 4 through 5 years with Duchenne muscular dystrophy who have a confirmed mutation in the dystrophin gene.

The supplemental application seeks to convert the accelerated approval to a full approval and expand the indication to treat Duchenne muscular dystrophy patients with a confirmed mutation in the DMD gene.

Elevidys is a one-time therapy that delivers a gene to muscle that codes for a shortened, functional form of dystrophin, which is mutated in Duchenne muscular dystrophy. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal. Duchenne affects about 1 in 3,500 to 5,000 males born worldwide.

Elevidys launched with a wholesale acquisition cost (WAC) of $3.2 million. A cost-effectiveness analysis sponsored by Sarepta indicated the value of Elevidys between $5 million and $13 million compared with standard of care alone, with a willingness-to-pay threshold of $500,000. The analysis found that Elevidys increases quality of life and equal value of life years gained, a measure used to determine how much a treatment can extend life.

FDA Sets Goal Date for Augtyro for Solid Tumors with NTRK Gene Fusions

The FDA has accepted for priority review Bristol Myers Squibb’s supplemental new drug application for Augtyro (repotrectinib) and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 15, 2024. Augtyro is being reviewed as a treatment for adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. If approved, it would be indicated for patients whose cancer is locally advanced or metastatic.

NTRK gene fusions can play a role in the development of cancer. They are rare in patients with solid tumors with less than 1% of patients testing positive, though may be more frequent in patients with other cancers.

Augtyro is an oral tyrosine kinase inhibitor (TKI) that was developed by Turning Point Therapeutic, which Bristol Myers Squibb acquired in 2022. In November 2023, the FDA approved Augtyro to treat adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer NSCLC. The wholesale acquisition cost (WAC) for an adult is $29,000 based on 30-day supply of 160 mg twice a day.

The supplemental filing is based on data from the TRIDENT-1 and CARE trials. In the TRIDENT-1 phase 1/2 study, Augtyro was studied in patients with advanced solid tumors, including non-small cell lung cancer and included patients with ROS1 or NTRK fusions.

FDA Grants Priority Review to Seladelpar as Second-line Treatment for Rare Liver Disease

The FDA has granted priority review for CymaBay’s seladelpar to treat adults with primary biliary cholangitis (PBC), including those with itch in patients without cirrhosis or those who are intolerant to ursodeoxycholic acid. The agency has set a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 14, 2024

PBC is a progressive autoimmune disease that results from the destruction of the bile ducts in the liver. This leads to inflammation, scarring and cirrhosis. The disease affects mostly women.

Seladelpar is an orally active PPARδ agonist that has demonstrated a statistically significant improvement in biochemical markers of disease progression. The FDA had updated the Breakthrough Therapy Designation for seladelpar in October 2023 in recognition of clinical data that indicated seladelpar may provide meaningful improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and improvement in pruritus (itching) in patients without cirrhosis or with compensated cirrhosis.

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