IntraBio Inc in the NEWS
IntraBio Inc. announced that the FDA has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
IntraBio Inc., a US biopharmaceutical company, focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich.
AQNEURSA is the only FDA-approved stand-alone therapy indicated for treating Niemann-Pick disease type C (NPC).
From IntraBio Inc and Other Experts
Mallory Factor, President and Chief Executive Officer of IntraBio said, “IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC, and today we celebrate a major milestone in this tremendous effort. Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that AQNEURSA may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to develop AQNEURSA for these additional indications rapidly.”
Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, commented, “The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C,” For too long, our community has been without an approved therapy for the treatment of NPC. Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”
The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial, which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients with a confirmed diagnosis of NPC.
The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving AQNEURSA. Results from the study showed AQNEURSA significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks.
These findings were published in the February 1, 2024, issue of the New England Journal of Medicine.
The primary outcome assessed by the FDA was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). SARA is a clinical assessment tool that assesses gait, stability, speech, and upper and lower limb coordination across eight individual domains. fSARA consists only of gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses.
AQNEURSA was well tolerated in the trial with the most common adverse reactions (incidence ≥5% and greater than placebo in Period I of the trial) being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Indication
AQNEURSA™ (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
For females of reproductive potential, verify that the patient is not pregnant before initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.
There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or the underlying maternal condition.
Adverse Reactions
The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
- Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.
- Monitor more frequently for P-gp substrate-related adverse reactions when used concomitantly with
AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.
AQNEURSA CaresTM Support for Patients
IntraBio offers support programs to eligible patients through their Patient Support Service, AQNEURSA CaresTM. This program includes financial support to reduce or eliminate out-of-pocket costs for qualifying patients and connects patients with third-party resources. AQNEURSA Cares includes access to financial and educational resources and a dedicated team of specialists. The team is available to help with individuals’ unique challenges including starting treatment, questions about taking the medication, and navigating insurance coverage. Contact 866-200-0419 to speak to an AQNEURSA Cares team representative to seek assistance with any questions or concerns about access to AQNEURSA.
Prohost Observations
The approval of the first treatment of Niemann-Pick disease type C is exciting news for the families of 100,000 live births and adults with this rare, inherited lysosomal disease. NPC patients experience systemic, neurological, and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives. The patients receiving AQNEURSA compared to placebo showed a greater improvement in fSARA score.
News & Comments
September 26, 2024
The FDA Approved IntraBio Product AQNEURSA As the only treatment for Niemann-Pick Disease Type C
IntraBio Inc in the NEWS
IntraBio Inc., a US biopharmaceutical company, focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich.
AQNEURSA is the only FDA-approved stand-alone therapy indicated for treating Niemann-Pick disease type C (NPC).
From IntraBio Inc and Other Experts
Mallory Factor, President and Chief Executive Officer of IntraBio said, “IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC, and today we celebrate a major milestone in this tremendous effort. Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that AQNEURSA may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to develop AQNEURSA for these additional indications rapidly.”
Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, commented, “The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C,” For too long, our community has been without an approved therapy for the treatment of NPC. Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”
The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial, which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients with a confirmed diagnosis of NPC.
The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving AQNEURSA. Results from the study showed AQNEURSA significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks.
These findings were published in the February 1, 2024, issue of the New England Journal of Medicine.
The primary outcome assessed by the FDA was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). SARA is a clinical assessment tool that assesses gait, stability, speech, and upper and lower limb coordination across eight individual domains. fSARA consists only of gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses.
AQNEURSA was well tolerated in the trial with the most common adverse reactions (incidence ≥5% and greater than placebo in Period I of the trial) being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Indication
AQNEURSA™ (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
For females of reproductive potential, verify that the patient is not pregnant before initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued.
There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or the underlying maternal condition.
Adverse Reactions
The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized.
U.S. full Prescribing Information for AQNEURSA is available at https://intrabio.com/wp-content/aqneursa-prescribing-information.pdf.
AQNEURSA CaresTM Support for Patients
IntraBio offers support programs to eligible patients through their Patient Support Service, AQNEURSA CaresTM. This program includes financial support to reduce or eliminate out-of-pocket costs for qualifying patients and connects patients with third-party resources. AQNEURSA Cares includes access to financial and educational resources and a dedicated team of specialists. The team is available to help with individuals’ unique challenges including starting treatment, questions about taking the medication, and navigating insurance coverage. Contact 866-200-0419 to speak to an AQNEURSA Cares team representative to seek assistance with any questions or concerns about access to AQNEURSA.
It’s important to read the Forward-Looking Statement.
Prohost Observations
The approval of the first treatment of Niemann-Pick disease type C is exciting news for the families of 100,000 live births and adults with this rare, inherited lysosomal disease. NPC patients experience systemic, neurological, and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives. The patients receiving AQNEURSA compared to placebo showed a greater improvement in fSARA score.
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