FDA Updates for Week of Oct. 10, 2022

In COVID-19 news, the FDA has authorized updated boosters for younger children. The FDA also approved an at-home heart failure therapy, but issued a CRL for a Parkinson’s therapy. The agency has assigned PDUFDA dates for several BLA, including a hemophilia A gene therapy and Eylea for retinopathy in premature infants. Regulators have also announced an Adderall shortage. Additionally, a nonprofit company has begun the application process for OTC version of naloxone.

FDA authorizes updated COVID-19 boosters for younger children.

The FDA has issued an emergency use authorization for both the Moderna and Pfizer updated COVID-19 vaccine boosters in children. These bivalent booster vaccines add the omicron variant BA.4 and BA.5 to the original SARS-CoV-2 and a component of omicron lineage BA.1.

Moderna’s updated booster is authorized for children six years of age and older. In August, the FDA had authorized Moderna’s updated booster for those 18 years and older. The BioNTech/Pfizer updated booster is authorized for children five years and older. In August, the agency had authorized the BioNTech/Pfizer updated booster for those 12 years and older.

For each of the bivalent COVID-19 vaccines authorized, the FDA relied on data that it had previously evaluated from a clinical study in adults of a booster dose of a bivalent COVID-19 vaccine that contained a component of the original strain of SARS-CoV-2 and a component of omicron lineage BA.1. In addition, the FDA has evaluated immune response and safety data from clinical studies of the monovalent mRNA COVID-19 vaccines, including as a booster dose in pediatric age groups.

FDA approves heart failure therapy that provides an alternative to hospital treatment.

The FDA has approved scPharmaceuticals’ Furoscix (furosemide injection) to treat adult patients with congestion due to fluid overload in chronic heart failure. It uses a proprietary formulation of furosemide delivered via an On-Body Infusor and delivers an 80-mg dose that is an equivalent of IV diuresis.

Furoscix enables subcutaneous administration at home by the patient or a caregiver with the use of the On-Body Infusor, which was developed using West Pharmaceutical Services SmartDose technology. Once the pre-filled cartridge is inserted into the pre-programmed infusor and attached to the abdomen, the device is activated to deliver the 80-mg dose over five hours.

Company officials said Furoscix will be launched in the first quarter of 2023, but indicated no information is available on the cost of the therapy. Medicare D will be the predominant payer segment for Furoscix, and a spokesperson said the company is focused on seven priority payer/PBM targets representing >85% of the Medicare lives payer landscape: United Health, Humana, CVS/Aetna, ESI/Cigna, Centene/WellCare, Anthem and Kaiser.

FDA issues CRL for Parkinson’s therapy.

The FDA has issued a complete response letter (CRL) for the Supernus Pharmaceuticals’ new drug application for SPN-830. SPN-830 is an apomorphine infusion device under review to treat short-term, intermittent “off” episodes in people with Parkinson’s disease. The infusion device would create a continuous treatment with apomorphine.

The FDA is asking for additional analysis related to the infusion device used with SPN-830. The agency is also requiring inspections of manufacturing facilities. The company had resubmitted the NDA in December 2021 following the agency’s initial refusal to file the application because it was incomplete.

FDA announces Adderall shortage

While pharmacies were already reporting Adderall (amphetamine mixed salts) shortages this summer, the FDA officially posted a drug shortage notice on October 12. Adderall treats both attention deficit hyperactivity disorder (ADHD) and narcolepsy.

“FDA is in frequent communication with all manufacturers of amphetamine mixed salts, and one of those companies, Teva, is experiencing ongoing intermittent manufacturing delays,” the FDA said in the notice. “Other manufacturers continue to produce amphetamine mixed salts, but there is not sufficient supply to continue to meet U.S. market demand through those producers.”

“Until supply is restored, there are alternative therapies including the extended-release version of amphetamine mixed salts available to healthcare professionals and their patients for amphetamine mixed salts’ approved indications,” the FDA said. “Patients should work with their healthcare professionals to determine their best treatment option.”

The FDA’s Adderall drug shortages web site lists the availability and unavailability of every presentation of the drug from each manufacturer. For example, Teva has many dosages available, but some sizes — including 12.5 mg and 15 mg 100 count — are on back order until March 2023.

FDA accepts BLA for hemophilia A gene therapy.

The FDA has accepted the resubmission of BioMarin Pharmaceutical’s biologics license application (BLA) for the AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. If approved, valoctocogene roxaparvovec would be the first gene therapy in the U.S. for the treatment of severe hemophilia A.

The Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2023, but company officials indicated the FDA may extend the review time to assess additional long-term follow-up data.

The company had resubmitted the BLA early in October 2022. The FDA had issued a complete response letter for valoctocogene roxaparvovec in August 2020 requesting two-year outcomes from the global GENEr8-1 phase 3 study and supportive data from five years of follow-up from the ongoing phase 1/2 dose escalation study.

FDA accepts sBLA for Eylea for retinopathy in premature infants.

The FDA has accepted for priority review the supplemental biologics license application (sBLA) for Eylea (aflibercept) injection to treat patients with retinopathy of prematurity (ROP) in preterm infants. The target action date for the FDA decision is Feb. 11, 2023. Retinopathy of prematurity is a leading cause of blindness in children, and each year between 1,100 and 1,500 infants develop the disease.

Developed by Regeneron, Eylea is currently available to treat patients with age-related macular degeneration, diabetic macular edema and diabetic retinopathy. It is a VEGF inhibitor that is injected into the eye. It is designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels in the eye.

Eylea is also under review for an additional dosing regimen with a longer-term interval between doses in patients with diabetic retinopathy. The target action date for this decision is Feb. 28, 2023.

Nonprofit company begins application process for OTC naloxone.

Harm Reduction Therapeutics, nonprofit pharmaceutical company, has initiated a rolling submission for a new drug application (NDA) for RiVive (naloxone), as a nonprescription emergency treatment for opioid overdose. The first module has been submitted to the FDA, and the company expects the submission to be complete by the end of the year.

RiVive is an HRT001 is an intranasal formulation of naloxone (3.0 mg) delivered as an atomized spray (0.1 ml) using a standard unit dose system for single administration. If approved, the company anticipates a launch of early 2024.

The NDA for RiVive is supported by a nonclinical program and a pivotal phase 1 relative bioavailability study demonstrating that RiVive produced a three-fold higher systemic exposure with comparable early absorption to the reference naloxone product. The company was founded in 2017 to develop a low-cost, OTC naloxone product