FDA Updates for Week of October 18, 2021

Additional COVID-19 vaccine boosters receive EUA; positive results for Pfizer booster.

The FDA issued an emergency use authorization (EUA) for Moderna’s COVID-19 vaccine booster dose for people aged 65 and older; people aged 18 to 64 who are at high risk of severe COVID-19; and people aged 18 to 64 with frequent institutional or occupational exposure to the virus.

The agency also authorized a single booster dose of the Moderna’s COVID-19 vaccine for people who have completed a primary vaccination with other authorized or approved COVID-19 vaccines.

The FDA issued an EUA for Johnson & Johnson’s COVID-19 vaccine as a booster for adults aged 18 and older at least two months following primary vaccination with single-shot Johnson & Johnson COVID-19 vaccine, and for eligible individuals who received a different authorized or approved COVID-19 vaccine.

The J&J booster will be the same formulation and dosage as the primary shot.

Meanwhile, Pfizer and BioNTech announced an efficacy rate of 95.6% for their COVID-19 vaccine booster. The phase 3 randomized, controlled study encompassed more than 10,000 individuals 16 years and older.

FDA approves novel therapy for macular degeneration.

The FDA has approved Genentech’s Susvimo (ranibizumab) for the treatment of patients with wet, age-related macular degeneration (AMD). Susvimo is a refillable ocular implant that delivers ranibizumab continuously, offering people living with wet AMD an alternative to anti-VEGF eye injections needed as often as once a month. The implant is surgically inserted into the eye during a one-time, outpatient procedure and refilled every six months.

Susvimo was generally well-tolerated, but the implant was associated with a three-fold higher rate of infections than monthly intravitreal injections of ranibizumab. In clinical trials, 2% of patients with an implant experienced at least one episode of endophthalmitis.

Many of these events were associated with conjunctival retractions or erosions. Conjunctival retraction is an opening or gaping in the layer (conjunctiva) that covers the white part of the eye, which may cause the implant to be exposed. Conjunctival erosion is an area that becomes missing in the conjunctiva. Both of these events may require surgery.

In clinical trials, the most common adverse events were conjunctival hemorrhage, conjunctival hyperemia, iritis and eye pain.

FDA approves Dupixent for children with asthma.

The FDA has approved Dupixent (dupilumab) as an add-on maintenance treatment of patients aged 6 to 11 years with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid-dependent asthma.

Developed by Regeneron Pharmaceuticals and Sanofi, Dupixent is the only biologic to improve lung function in children 6 to 11 years old.

FDA approves Tyrvaya for dry eye disease

The FDA has approved Tyrvaya (varenicline solution) Nasal Spray 0.03 mg for the treatment of the signs and symptoms of dry eye disease. It is the first nasal spray approved for this indication.

The therapy, developed by Oyster Point, is believed to bind to cholinergic receptors to activate the trigeminal parasympathetic pathway resulting in increased production of basal tear film as a treatment for dry eye disease.

Tyrvaya is a highly selective cholinergic agonist delivered twice daily as an aqueous nasal spray into each nostril to activate basal tear production. Nasal spray administration provides a new way to treat dry eye disease. Nasal delivery may allow some patients who have difficulty independently administering topical eye drops.

Tyrvaya will be available in November 2021 in cartons containing two multidose nasal spray bottles. Each nasal spray bottle covers treatment for 15 days, administered twice daily into each nostril.

FDA approves Biktarvy for young children with HIV.

The FDA has approved a new low-dose tablet dosage form of Gilead’s Biktarvy (bictegravir 30 mg/emtricitabine 120 mg/tenofovir alafenamide 15 mg) for pediatric patients living with HIV-1 infection.

The approval is based on data from a cohort of patients in a phase 2/3 study, which found Biktarvy low-dose tablets to be effective and generally well-tolerated through 24 weeks in virologically suppressed children living with HIV-1. Cohort 3 enrolled 22 participants weighing between 14 kg and 25 kg who continued on treatment for 48 weeks and could then continue to receive study drug through an extension phase.

Nearly 3 million children worldwide under the age of 19 are living with HIV. An estimated 120,000 children and adolescents died from AIDS-related causes in 2020. About 72% of these deaths occurred among children under 10 years old.

FDA approves ZIMHI to treat opioid overdose.

The FDA has approved ZIMHI (naloxone hydrochloride) injection as an additional option to treat opioid overdose. ZIMHI is administered using a single-dose, prefilled syringe that delivers 5 mg of naloxone hydrochloride solution through intramuscular or subcutaneous injection. It was developed by Adamis Pharmaceuticals.

ZIMHI received approval through the 505(b)(2) approval pathway, which allows review of FDA’s finding that a previously approved drug is safe and effective or on published literature to support the safety and/or effectiveness of the proposed product, if such reliance is scientifically justified. In this case, Adamis’ application relied, in part, on the FDA’s finding of safety and effectiveness for Narcan injection to support approval.

FDA approves generic of Revlimid.

The FDA has approved Dr. Reddy’s abbreviated new drug application for lenalidomide capsules, in 2.5 mg and 20 mg strengths, and given a tentative approval for 5 mg, 10 mg, 15 mg, and 25 mg strengths. Lenalidomide is a therapeutic equivalent generic version of Celgene’s Revlimid, which is an oral cancer drug that is used for the treatment of multiple myeloma.

With this approval, Dr. Reddy’s is eligible for 180 days of generic drug exclusivity for lenalidomide capsules, 2.5 mg and 20 mg.

In September 2020, Dr. Reddy’s announced a settlement agreement of their litigation with Celgene, a wholly-owned subsidiary of Bristol Myers Squibb, relating to patents for the branded drug. Celgene agreed to provide Dr. Reddy’s with a license to sell volume-limited amounts of generic lenalidomide capsules in the United States beginning on a date sometime after March 2022.

Dr. Reddy’s is also licensed to sell generic lenalidomide capsules in the United States without volume restrictions beginning on Jan. 31, 2026.

FDA issues complete response letter for Tyvaso DPI.

The FDA has issued a complete response letter for United Therapeutics’ new drug application for Tyvaso DPI (treprostinil), for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.

The regulatory agency noted a deficiency related to an open inspection issue at a third-party facility that performs analytical testing of treprostinil drug substance.

Tyvaso DPI is a drug-device combination therapy comprised of a dry powder inhalation formulation of treprostinil. It was developed through a collaboration with MannKind, which developed the inhalation technology that is also used in MannKind's Afrezza (insulin human) inhalation powder product.

A nebulized formulation of Tyvaso is already available to treat pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.

Company officials expect the DPI version to be available by the summer of 2022.