Ingrezza for Huntington’s
The FDA has approved valbenazine (INGREZZA®) Sprinkle for the treatment of adults with tardive dyskinesia and chorea associated with Huntington’s disease. This new oral granules formulation provides an alternative administration option for those who experience dysphagia or have difficulty swallowing, offering the same dosage strengths as the original capsule form which can be sprinkled on soft food for easier intake.
Valbenazine, initially approved for these indications in its capsule form, is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor. It is designed to inhibit the release of dopamine, a neurotransmitter involved in controlling movement, thereby reducing uncontrollable movements associated with these disorders.
Tardive dyskinesia is characterized by involuntary, repetitive movements primarily affecting the face and upper body, often resulting from long-term use of antipsychotic medications. Huntington’s disease is a genetic neurodegenerative disorder causing motor dysfunction, cognitive decline, and psychiatric symptoms, with chorea being one of its primary movement disturbances.
The FDA approval of valbenazine in this new formulation was based on data demonstrating its bioequivalence and tolerability compared to the original INGREZZA capsules. The approval of valbenazine was granted to Neurocrine Biosciences, Inc.
Tivdak for Cervical Cancer
The FDA has approved tisotumab vedotin-tftv (TIVDAK) for adult patients with recurrent or metastatic cervical cancer who have experienced disease progression on or after chemotherapy. This approval represents a conversion to full approval from accelerated approval, which was initially granted in September 2021 based on tumor response and durability from the innovaTV 204 study. Tisotumab vedotin-tftv, an antibody-drug conjugate (ADC), functions through a mechanism where it binds to tissue factor (TF) on cancer cells, leading to the internalization of the ADC-TF complex and subsequent release of the cytotoxic agent monomethyl auristatin E (MMAE) that disrupts microtubules, causing cell death.
The full approval was based on results from the global, randomized Phase 3 innovaTV 301 clinical trial, which demonstrated a significant overall survival benefit of tisotumab vedotin compared to chemotherapy. The trial reported a 30% reduction in the risk of death and a median overall survival of 11.5 months for patients treated with TIVDAK versus 9.5 months for those receiving chemotherapy.
The approval of tisotumab vedotin was granted to Pfizer and Genmab A/S.
Xolremdi for WHIM
The FDA has approved mavorixafor (Xolremdi) for WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) in patients 12 years of age and older to increase the number of circulating mature neutrophils and lymphocytes.
This is the first approval for mavorixafor, a small molecule CXCR4 antagonist that acts by blocking the receptor to enhance the release of neutrophils and lymphocytes from the bone marrow.
WHIM syndrome, a rare genetic disorder, impairs immune function by reducing circulating mature neutrophils and lymphocytes, critical for combating infections. It has a very low incidence rate, occurring in about 1 in 5 million live births, and is associated with a high risk of recurrent and potentially life-threatening bacterial and viral infections.
The approval was based on findings from a 52-week, randomized, double-blind, placebo-controlled trial (NCT03995108), demonstrating that mavorixafor significantly improved absolute neutrophil and lymphocyte counts. The treatment also notably reduced the infection score by 40% compared to placebo, though it did not improve warts.
The approval of mavorixafor was granted to the manufacturer, X4 Pharmaceuticals.
Esprit Stent for CLTI-BTK
The FDA has approved the Esprit™ BTK Everolimus Eluting Resorbable Scaffold System (Esprit BTK System) for chronic limb-threatening ischemia (CLTI) below-the-knee (BTK). This is the first approval of its kind for a device in this category in the U.S., as previous standards of care were limited to balloon angioplasty, which often resulted in recurrent blockages and additional treatments.
The Esprit BTK System utilizes a novel approach with a dissolvable stent similar to dissolving sutures, designed to support vessel healing and keep arteries open. It gradually dissolves after providing necessary support for about three years, ideally leaving a healed and functional vessel.
Chronic limb-threatening ischemia is a severe form of peripheral artery disease (PAD), characterized by blocked arteries and reduced blood flow to the lower limbs, leading to significant pain, non-healing wounds, and sometimes necessitating amputation. CLTI has a notably poor prognosis.
The approval was based on findings from the LIFE-BTK trial, which showed that the Esprit BTK System significantly reduced disease progression and improved medical outcomes compared to the current standard care. The approval of the Esprit BTK System was granted to Abbott Laboratories.