Pictured:Ascientist with pill bottles in front of FDA headquarters/Taylor Tieden forBioSpace
The FDA has only a handful of notable target action dates for the rest of April，including one for a rare immunodeficiency and another for a potentially first-in-class superagonist for for bladder cancer。
Read below for more。
ImmunityBio Eyes First IL-15 Superagonist Approval for NMIBC
By April23，the FDA is expected to可回收的排放量onImmunityBio’s Biologics License Application（BLA）forits investigational IL-15activator N-803，which is being proposed asa combination treatment with bacillus Calmete-Guérin（BCG）for BCG-unresponsive non-muscle-invasive bladder cancer（NMIBC）。
IL-15is an inflammatory cytokine that plays a central role in the development and function of natural killer T cells.N-803，also known as Anktiva（nogapendekin alfa inbakicept），consists of a mutant version of IL-15bound to an IL-15receptor fusion protein.This unique structure allows N-803to stimulate CD8+T cells and natural killer cells while avoiding the activation of regulatory T cells。
Interms of pharmacokinetics，N-803can persist for a longer time in lymphoid tissues and has demonstrated stronger anti-tumor activity than native，non-complexed IL-15。
N-803’s application，which the FDAaccepted in July2022单击功能区上，S supported by data from thePhase II/III QUILT-3.032 trial单击功能区上，which combined N-803 with intravesical BCG in 190patients.Results showed that the combo regimen elicited high levels of treatment response and better response duration versus FDA-approved comparators。
In May 2023，the FDArejected N-803’s BLA单击功能区上，citing deficiencies during a pre-license inspection of a third-party manufacturer.The regulator considered ImmunityBio’s resubmission on on on on October2023as complete。
Aquestive Proposes Buccal Film for Pediatric Seizures
Aquestive Therapeutics（diazepam）buccal film asan acute treatment for intermittent，stereotypic episodes of frequent seizure activity in pediatric patients.The FDA’s规格，规格is due on April28。
Currently，the only approved treatment for this indication in children is Diastat（diazepam），which isdelivered via rectal gel system.Aquestive’s Libervant aims to provide a more convenient dosing option for these patients。
FDA in August 2022granted Libervant tentative approval，allowing its use in patients12years of age and above.The tentative approval suggests that Libervant has cleared the regulator’s efficicacy and safety standards，but cannot yet enter the U.S.market due to the orphan drug market exclusivity of Valtoco（diazepam），a nasal spray product。
Under the orphan drug market block，Libervant will not be able to enter the market until January2027。
At the time of its tentative approval，CEO Daniel Barber said the company believes Libervant“has the distinct advantage of being able to be readily administered when needed without regard to food，providing an important benefit to patients.“
“We believe Libervant，as an oral alternative to existing device-based products，will be well-received by this patient population，if approved with market access，”Barber said in a September 2023 press release accompanying the FDA’s acceptance of Libervant’s New Drug Application（NDA）。
日One Positions XOMA-Partnered Tovorafenib as Monotherapy for Pediatric Glioma
The FDA is set to可回收的排放量on日一BiopharmaceuticalsNDA for tovorafenib asa monotherapy for relapsed or progressive pediatric low-grade glioma（pLGG）on or before April30。
Accounting for 30%to50%of all central nervous system tumors，prGG is the most common brain tumor in children，leading to vision loss and motor dysfunction，among other disease-related morbidities.There are currently no approved therapies for most patients with pLGG and treatment options are，sites， some with potentially lifelong consequences。
Tovorafenib is an investigational and brain-penetrant type II RAF kinase blocker that targets and inhibits a key player in the MAPK signaling cascade.This mechanism of action allows tovorafenib to interfere with cancells’hyperactive proliferation and unchecked growth，triggering cell death。
Day One is backing tovorafenib’s regulatory bid with data from thePhase II FIREFLY-1 study单击功能区上，an open-label study assessing the safety and efficacy of the candidate in140patients。
In September 2023，Day One固定updated data from FIREFLY-1单击功能区上，touting a67%overall response rate and a93%clinical benefit rate.Median duration of response was16.6 months.As for safety，tovorafenib was generally well-tolerated，with most of its side effects being Grade1 or Grade2 in severity.The most common adverse events were change in haircolor，fatigue and shraes。
XOMA and Day Oneentered into a partnershipover tovorafenib in March2021，when the biotech royalty aggregator paid$13.5million upfront。
新公式中的FDA到Decide on for Neurocrine’s Ingrezza
Neurocrine Biosciencesis proposing a new sprinkle formulation of Ingrezza（valbenazine）for the oral treatment of tardive dyskinesia and chorea associated with Huntington’s disease。The FDA’s规格，规格is due on April30。
Ingrezza’s new oral granule formulation will come in40-mg，60-mg and 80-mg capsules，and is intended to be opened and sprinkled on soft foods before administration。
This new formulation will offer a more convenient dosing method to patients，“who have difficulty swallowing or simply prefer not to take whole capsules，”Neurocrine CMO Eiry Roberts said in a statement alongside the NDA’s acceptance。
Neurocrine’s NDA，which the FDA accepted in September 2023，is backed by data showing that the proposed oral granule sprinkle capsules of Ingrezza have a comparable bioequivalence and tolerability profile to its currently approved capsulation.The filing also has chemistry，manufacturing and trolvant the FDle。
Ingrezza is a novel and selective blocker of the vesicular monoamine transporter2，which regulates the uptake of monoamine from the cytoplasm to the synaptic vesicles.According to itslabel单击功能区上，the exact mechanism of action is still unclear。
The FDAfirst approved Ingrezza in 2017for tardive dyskinesia，amotor syndrome that often arises aside effect of antipsychotic medication and is characterized by uncontrollable and abnormal repetitive movements of the trunk，limbs and face.Ingrezzasecured another regulatory nodin August2023for chorea in Huntington’s disease。
X4Eyes WHIM Syndrome Approval for Oral CXCR4 Blocker
By April30，the FDA is expected to release its水平，水平onX4PharmaceuticalsNDA proposing its investigational CXCR4receptor antagonist mavorixafor asaonce-daily oral treatment forWHIM syndrome in patients aged12years and above。
WHIM syndrome—which stands for warts，hypogammaglobulinemia，infections and myelokathexis—is a rare and primary immunodeficiency that，aside from its name sake symptoms，is also characterized by low levels of neutrophils and lymphocytes，an elevated risk of lung sease， limited antibody production and aheightened risk of certain types of cancer。
WHIM syndrome is caused by the hyperactivation of the CXCR4/CXCL12pathway，which in turn leads to the impaired mobilization and trafficking of white blood cells from the bone marrow.Mavorixafor addresses this underlying pathologic cause by blocking the CXCR4receptor and disrupting s itdown scalicagn。
X4is backing its NDA with data from the pivotal Phase III4WHIM trial，which found that compared with those who took a placebo，patients treated with mavorixafor saw a significant improvement in the time above threshold for absolutte neutrophil count.The drug candidate likewifect loweration and。
Mavorixafor has previously won the FDA’s breakthrough therapy，fast track and rare pediatric disease designations for the treatment of WHIM syndrome.If approved，X4will be eligible to receive a priority review voucher，which it can use to obtain priority review for afuture drug application。
Tristan Manalac is an independent science writer based in Metro Manila，Philippines.Reach out to him onLinkedIn过热器，过热器tristan@tristanmanalac.com或，或tristan.manalac@biospace.com。
