Here is information on the latest US FDA approvals, the week of April 8 – April 12, 2024
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Fasenra Pediatric Asthma Expansion
The FDA has approved benralizumab (Fasenra) for add-on maintenance treatment for patients with severe asthma aged 6 to 11 with an eosinophilic phenotype. This follows the initial approval of benralizumab in 2017 as an add-on maintenance treatment for severe eosinophilic asthma in patients aged 12 and older. Benralizumab is an interleukin-5 receptor alpha-directed cytolytic monoclonal antibody that reduces eosinophilic inflammation, a key factor in the pathophysiology of severe asthma.
Severe asthma is a complex condition that significantly impacts quality of life, particularly in children, and can lead to frequent exacerbations and hospitalizations. This condition often requires more intensive management strategies compared to milder forms of asthma.
The latest approval was based on findings from the TATE study, an open-label, multinational, non-randomized, parallel assignment Phase 3 trial. In this trial, the pharmacokinetics (PK) and pharmacodynamics (PD) of benralizumab in children aged 6 to 11 years old with severe eosinophilic asthma were consistent with those observed in prior trials in older age groups. The safety and efficacy profiles were also aligned with previous findings. The approval of benralizumab was granted to AstraZeneca.
https://www.astrazeneca-us.com/media/press-releases/2024/fasenra-approved-for-treatment-of-children-aged-6-to-11-with-severe-asthma.html
Xcopri New Delivery Methods
The FDA has approved two new administration methods for cenobamate tablets (Xcopri) for adult patients with partial-onset seizures. These new methods include crushing the tablets to mix with water for oral administration or administration via a nasogastric tube.
Cenobamate was first approved in 2019 as an antiseizure medication (ASM). It acts by reducing repetitive neuronal firing, primarily through inhibiting voltage-gated sodium currents, though it is also a positive allosteric modulator of the γ-aminobutyric acid ion channel. The drug can be used alone or in combination with other ASMs.
Partial-onset seizures, the condition for which cenobamate is indicated, are a form of epilepsy where seizures begin in one area of the brain and can remain localized or spread. Managing this condition effectively is critical as it can significantly impact patient quality of life.
The approval of these new administration methods was based on findings from an open-label, randomized, single-center, 3-period, 6-sequence, balanced crossover study. This study demonstrated bioequivalence between the new administration routes and the original method of swallowing an intact tablet. The approval of cenobamate for these methods was granted to SK Biopharmaceuticals.
https://www.prnewswire.com/news-releases/xcopri-cenobamate-tablets-cv-receives-fda-approval-for-alternate-methods-of-administration-that-include-crushed-tablet-in-liquid-suspension-taken-orally-or-through-a-nasogastric-tube-302113538.html
New MRD Endpoint for Myeloma
The FDA’s Oncologic Drug Advisory Committee unanimously recommended the adoption of minimal residual disease (MRD) as a new endpoint for the accelerated approval of treatments for multiple myeloma. Advisory committee votes usually but not always influence the FDA’s final decisions.
Multiple myeloma, a type of blood cancer characterized by malignant plasma cells accumulating in the bone marrow, often leads to extensive bone damage and renal failure among other complications. Traditional endpoints for drug approval, like overall response rates, require lengthy observation periods and often only show a partial picture of a patient’s response to therapy. In contrast, MRD can provide a more immediate and sensitive measure of disease presence at very low levels, potentially allowing for quicker and more adaptive treatment strategies.
The proposed adoption of MRD as a new endpoint could reshape the landscape of multiple myeloma treatment, enabling faster introduction of new therapies and possibly improving patient outcomes. The approval of this new endpoint for accelerated drug approval processes is now pending further FDA review.
Mpox PCR Test Home Collection Kit
The FDA has granted Emergency Use Authorization (EUA) for Labcorp’s Mpox PCR Test Home Collection Kit for the diagnosis of non-variola Orthopoxvirus infection, including monkeypox virus that causes mpox, in patients 18 years of age or older suspected of mpox infection. This marks the first authorization for an at-home mpox collection kit
The Mpox PCR Test Home Collection Kit employs polymerase chain reaction (PCR) technology to detect the DNA of the non-variola Orthopoxvirus in lesion swab samples collected at home by patients.
Monkeypox, also known as mpox, is a viral disease characterized by a rash and flu-like symptoms. It is less severe but similar to smallpox, which was eradicated in 1980. The recent increases in mpox cases in the United States have heightened the need for accessible diagnostic options to manage the spread and treatment of the virus.
The approval of the Mpox PCR Test Home Collection Kit was granted to Labcorp.
https://www.prnewswire.com/news-releases/labcorp-receives-fda-emergency-use-authorization-for-mpox-pcr-test-home-collection-kit-302113019.html
Dovato for Adolescents With HIV
The FDA has approved dolutegravir/lamivudine (Dovato) for adolescents living with HIV. This approval introduces Dovato as the first and only oral, two-drug, single-tablet regimen available for individuals aged 12 and older with HIV.
Dovato combines dolutegravir, an integrase strand transfer inhibitor (INSTI), with lamivudine, a nucleoside reverse transcriptase inhibitor (NRTI). Dolutegravir works by binding to the integrase active site and blocking the strand transfer step of retroviral DNA integration, which is crucial for the HIV replication cycle. Lamivudine inhibits reverse transcriptase through DNA chain termination, interrupting the viral replication process.
HIV in adolescents presents unique treatment challenges, including the need for lifelong therapy. Simplifying treatment regimens can be crucial for adherence and overall management of the disease.
The approval of dolutegravir/lamivudine was based on findings from the DANCE study, which assessed the regimen in treatment-naïve adolescents, as well as data from adult trials, including GEMINI-1, GEMINI-2 (treatment-naïve adults), and TANGO (treatment-experienced adults). The DANCE study showed that a significant proportion of participants aged 12 to 18 years achieved and maintained viral suppression at Week 48, with safety and efficacy profiles similar to those observed in adults. The approval of dolutegravir/lamivudine was granted to ViiV Healthcare.
https://viivhealthcare.com/en-us/media-center/news/press-releases/2024/april/fda-approval-of-dovato/