Home Cellular health FDA updates for the week of October 3, 2022

FDA updates for the week of October 3, 2022


The FDA uses the real world to show that Boostrix prevents infections in infants. The agency also approves a label extension for Oxlumo, a new delivery method for Trogarzo, and accepts an sBLA for Takhzyro. In COVID-19 news, Eiger will not be submitting EUAs for COVID-19 treatment. Additionally, an OIG report reveals that many accelerated approvals have delayed confirmatory trials.

Real-world data confirms that Boostrix used during the third trimester prevents infections in infants.

The FDA has approved Boostrix for immunization during the third trimester of pregnancy to prevent whooping cough, commonly known as whooping cough, as a way to prevent infection in infants under two months of age.

Boostrix is ​​composed of tetanus toxoid, reduced diphtheria toxoid and acellular pertussis vaccine (Tdap). It was originally approved by the FDA in 2005 as a single dose booster vaccination against tetanus, diphtheria, and pertussis in people ages 10 to 18. Later, the FDA also approved Boostrix to include use in people 19 years of age and older and to include use of an additional dose 9 years or older after the initial dose of a Tdap vaccine.

FDA approval of Boostrix has always included its use during pregnancy to protect the vaccinated person. Today’s approval is specific for use during pregnancy to prevent whooping cough in infants less than 2 months old. Since 2012, the CDC has recommended the use of Tdap vaccines during the third trimester of each pregnancy.

Regulators evaluated the effectiveness of Boostrix in preventing pertussis in infants using a new analysis of data from an observational case-control study of the effectiveness of the Tdap vaccine.

FDA approves label extension for Oxlumo.

The FDA has approved a Supplemental New Drug Application for Oxlumo (lumasiran) from Alnylam Pharmaceuticals to treat patients with primary hyperoxaluria type 1 (PH1), a rare and serious metabolic disease that often manifests as kidney stones. It is caused by mutations in the AGXT gene that lead to the buildup of oxalate, which is filtered out by the kidneys. The disease affects fewer than 5,000 people in the United States.

Oxlumo is an RNAi therapy administered by subcutaneous injection and is indicated to lower plasma oxalate levels and urinary oxalate levels in pediatric and adult patients. This authorization adds to the indication the reduction of plasma oxalate levels. The therapy harnesses RNA interference, a natural cellular process of gene silencing. The FDA approved Oxlumo in November 2020 for the treatment of PH1 to reduce urinary oxalate levels in pediatric and adult patients.

FDA approves new delivery method for Trogarzo in HIV.

The FDA has approved Thera Technologies’ “push” administration of Trogarzo (ibalizumab-uiyk) to treat patients with human immunodeficiency virus type 1 (HIV-1) infection in highly experienced adults. Trogarzo has been approved for administration by intravenous (IV) push, a method in which undiluted medicine is pushed through a syringe for faster delivery into the body’s circulation. This method reduces the maintenance dose from a 15-minute IV infusion to an undiluted 30-second IV injection every two weeks.

The FDA originally approved Trogarzo, a long-acting monoclonal antibody, in March 2018 to be administered intravenously as a single loading dose followed by a maintenance dose every two weeks. Trogarzo is also being studied for administration by intramuscular injection in the continuation of the TMB-302 study. The study is now fully enrolled, with the last patient visit scheduled for November 2022.

FDA accepts BLA for Takhzyro in young children with HAE.

The FDA has accepted a Supplemental Biologics License Application (sBLA) for the potential expanded use of Takeda’s Takhzyro (lanadelumab-flyo) to prevent attacks of hereditary angioedema (HAE) in pediatric patients aged 2 at

Takhzyro is currently approved in patients 12 years of age and older to prevent attacks of hereditary angioedema (HAE), a rare disease that causes recurrent episodes of swelling of the limbs, face, intestinal tract and airways. Symptoms of hereditary angioedema usually begin in childhood and worsen during puberty. On average, untreated people have an attack every one to two weeks, and most episodes last about three to four days.

Eiger will not submit EUAs for COVID-19 treatment.

One month after the FDA said it required additional data on Eiger BioPharmaceuticals’ Emergency Use Authorization (EUA) request of peginterferon lambda to treat patients with mild to moderate COVID-19, the pharmaceutical manufacturer said it would not submit the EUA.

Following a pre-EUA information exchange with the FDA regarding the TOGETHER Phase 3 study of peginterferon lambda for COVID-19, the agency indicated in September that it was not yet in a position to determine whether the criteria for submitting an application and issuing an EUA are likely to be met, Eiger said in a press release at the time.

Eiger officials said they are evaluating the next steps for this program in the United States, as well as ex-US emergency use authorization pathways and strategic options for further development of peginterferon lambda. for COVID-19 and other viral respiratory infections.

Eiger has secured worldwide rights to peginterferon lambda, a first-in-class investigational late-stage interferon type 3 (IFN) that stimulates immune responses essential for the development of host protection during viral infections, by Bristol-Myers Squibb.

OIG: One-third of accelerated approvals delayed confirmatory testing.

Medicare and Medicaid spent more than $18 billion from 2018 to 2021 on 18 drugs that were granted fast-track approval and had incomplete confirmatory trials after their originally scheduled completion date, according to new analysis from the Bureau of the Inspector General of the United States Department of Health and Human Services. The OIG estimated that Medicare Part B and Part D spent more than $14 billion, and Medicaid expenditures—both for fee-for-service and managed care—for these drugs amounted to nearly of $3.6 billion.

OIG officials said the review stemmed from concern that the regulator’s oversight of fast-track approval was lax. Accelerated approvals often use surrogate endpoints that predict clinical benefit, but do not measure clinical benefit. The pathway is meant to provide earlier access to drugs to treat serious illnesses. Companies are required to conduct confirmatory trials to verify clinical benefits and provide a timeline for completion.

Since the accelerated approval pathway began in 1992, 278 drug applications have been granted accelerated approval by the FDA’s Center for Drug Evaluation and Research (CDER). Of these applications, 104 have incomplete confirmatory trials. Of those 104, 34% (35 of 104) have at least one trial past its originally scheduled completion date. Additionally, 13% of all expedited drug approval requests have been withdrawn, half of them since January 2021.

Four drug applications have confirmatory trials that are significantly overdue — ranging from more than five years to nearly 12 years past their original completion dates. OIG researchers found that of these four drugs that were significantly behind confirmatory trials, proamatin (midodrine hydrochloride) had the highest estimated Medicare Part D expenditures at $142 million and Makena ( hydroxyprogesterone caproate) had the highest estimated Medicaid expenditures, nearly $700 million.