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FDA Updates for the Week of Nov. 14, 2022


In a busy week, the FDA approved the first drug to delay diabetes, a second interchangeable Lantus biosimilar, and an alternate dosing schedule for Rylaze. The agency also granted accelerated approval of a novel ovarian cancer therapy, updated the target date for pegcetacoplan NDA review, and proposed prescription-to-OTC naloxone. In addition, an advisory committee supported the use of Xphozah in CKD.

FDA approves first drug that delays diabetes.

The FDA has approved Provention Bio’s Tzield (teplizumab-mzwv) injection to delay the onset of stage 3 type 1 diabetes. It is indicated for adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes. Tzield is anti-CD3-directed antibody. It binds to CD3, a cell surface antigen present on T lymphocytes, and is believed to deactivate pancreatic beta cell autoreactive T lymphocytes. Tzield is administered by intravenous infusion once daily for 14 consecutive days.

Tzield will be available by the end of the year at a wholesale acquisition cost of $13,850 per vial for 14-day treatment course list price of $193,900, company executives said in a call with investors. The pricing was based on the value that it brings to patients.

The approval was based on a placebo-controlled trial with 76 patients with stage 2 type 1 diabetes. The trial results showed that over a median follow-up of 51 months, 45% of the 44 patients who received Tzield were later diagnosed with stage 3 type 1 diabetes, compared with 72% of the 32 patients who received a placebo. The mid-range time from randomization to stage 3 type 1 diabetes diagnosis was 50 months for the patients who received Tzield and 25 months for those who received a placebo.

FDA approves second interchangeable Lantus biosimilar.

The FDA has approved the second interchangeable biosimilar insulin product to Lantus (insulin glargine). Eli Lilly’s Rezvoglar (insulin glargine-aglr) is a long-acting human insulin analog indicated to improve glycemic control in adults and pediatric patients with diabetes. FDA previously approved Rezvoglar as a biosimilar to Lantus on Dec. 17, 2021. An interchangeable status now allows Rezvoglar to be substituted for Lantus at the pharmacy level.

Rezvoglar is available in 3 mL prefilled pens and is administered subcutaneously once daily. It may cause serious side effects, including hypoglycemia, severe allergic reactions, hypokalemia and heart failure.

Rezvoglar is the second approved interchangeable biosimilar insulin in the United States. This biosimilar joins Viatris’ Semglee (insulin glargine-yfgn), the first approved interchangeable biosimilar to Lantus, that FDA approved on July 28, 2021. Payer acceptance of Semglee has been uneven. Prime Therapeutics and Express Scripts have added Semglee to their preferred formularies, but OptumRx has excluded Semglee.

FDA grants accelerated approval of novel ovarian cancer therapy.

The FDA has granted accelerated approval for ImmunoGen’s Elahere (mirvetuximab soravtansine-gynx) to treat adult patients with epithelial ovarian, fallopian tube, or primary peritoneal cancer. It is indicated for women who have received one to three prior systemic treatment regimens. Elahere is a first-in-class antibody-drug conjugate targeted against folate receptor alpha, a cell-surface protein highly expressed in ovarian cancer, and is the first FDA approved ADC for platinum-resistant disease. About 35% to 40% of ovarian cancer patients express high levels of folate receptor alpha.

The wholesale acquisition cost is $6,220 for one vial. Patients typically undergo the use of three to four vials per cycle. This translates to $18,660 to $24,800 per treatment cycle.

The FDA has also granted approval of the Roche’s Ventana FOLR1 RxDx Assay, a companion diagnostic to aid in identifying patients eligible for treatment with Elahere.

Elahere was approved based on objective response rate and duration of response data from the pivotal SORAYA trial, a single-arm study in 106 patients with platinum-resistant ovarian cancer. Elahere objective response rate by investigator of 31.7%, including five complete responses. The median duration of response was 6.9 months. The safety of Elahere has been evaluated in a pooled analysis from three studies among a total of 464 patients. The label includes a boxed warning for ocular toxicity, including visual impairment, keratopathy, dry eye, photophobia, eye pain, and uveitis.

FDA approves alternate dosing schedule for Rylaze.

The FDA has approved Jazz Pharmaceuticals’ biologics license application (sBLA) for an additional dosing regimen for Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) to treat acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adults and children who have developed hypersensitivity to E. coli-derived asparaginase.

Rylaze was initially approved in July 2021 as administration every 48 hours. This approval adds a Monday/Wednesday/Friday intramuscular dosing schedule. At the time of it launch last year, Rylaze’s wholesale acquisition price was $4,390 per vial.

The new dosing option was based on data from the intramuscular administration part of the phase 2/3 trial. Results show that a dosing regimen of 25 mg/m2 administered intramuscularly on Monday morning and Wednesday morning, and 50 mg/m2 administered on Friday afternoon demonstrated a positive benefit-to-risk profile, with more than 90% of the patients achieving nadir serum asparaginase activity. No new safety signals observed in the trial.

FDA advisory committee supports Xphozah in CKD.

An FDA advisory committee has voted in favor of Ardelyx’s Xphozah (tenapanor) in the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. The Cardiovascular and Renal Drugs Advisory Committee voted 9 to 4 in favor of Xphozah as a monotherapy and 10 to 2 in favor of Xphozah in combination with phosphate binders.

Hyperphosphatemia is a serious condition resulting in an abnormally elevated level of phosphorus in the blood that is estimated to affect the vast majority of the 550,000 patients in the United States with chronic kidney disease on dialysis. When kidney function is impaired, phosphorus is not adequately eliminated from the body. Hyperphosphatemia is a major cause of morbidity and mortality in patients with chronic kidney disease.

FDA updates target date for pegcetacoplan.

The FDA has accepted Apellis Pharmaceuticals’ major amendment to the new drug application (NDA) for intravitreal pegcetacoplan to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The updated Prescription Drug User Fee Act (PDUFA) goal date is Feb. 26, 2023.

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is designed to be injected into the back of the eye. Geographic atrophy is an advanced form of age-related macular degeneration and a leading cause of blindness that impacts more than 5 million people worldwide, including 1 million people in the United States. There are currently no treatments for geographic AMD.

FDA proposes prescription-to-OTC naloxone.

The FDA issued a Federal Register notice, Safety and Effectiveness of Certain Naloxone Hydrochloride Drug Products for Nonprescription Use, that may help facilitate the development and approval of certain over-the-counter (OTC) naloxone drug products — including through the switch of certain naloxone drugs from prescription status to nonprescription status.

Naloxone is a medicine that can help reduce opioid overdose deaths and when administered timely — usually within minutes of the first signs of an opioid overdose — can counter the overdose effects, the agency said in a news release.

The Federal Register notice includes a preliminary assessment that certain naloxone drug products — up to 4 mg nasal spray and up to 2 mg autoinjector for intramuscular or subcutaneous use — may be approvable as safe and effective for nonprescription use. FDA’s notice does not cover all naloxone products, as more data are needed on the safety and efficacy for nonprescription use of higher dose naloxone products and naloxone supplied in other presentations (including vials, ampules or syringes without integrated needles) before a preliminary assessment with respect to those products can be reached, the agency said.

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