Saturday, July 13, 2024

Deficiency Focus: FDA Grants Fast Track Designation to Inozyme Pharma’s INZ-701 for ABCC6 Patients

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Deficiency in ABCC6 patients has led to the Food and Drug Administration (FDA) granting Fast Track Designation to Inozyme Pharma’s INZ-701, recognizing its potential as a promising treatment specifically designed to address this genetic condition. The Fast Track designation is based on preliminary safety and efficacy data from ongoing trials, and it aims to expedite the development and review process to bring this important therapy to patients more quickly.

This designation was based on preliminary safety and efficacy data from the ongoing Phase I/II trial of INZ-701 in adults with ABCC6 Deficiency, along with nonclinical pharmacology data. Inozyme Pharma believes that this Fast Track status will facilitate more frequent engagement with the FDA and expedited regulatory review.

FDA Fast Tracks INZ-701 for ABCC6 Deficiency, Paving Way for Pediatric Treatment Approval

“Through Fast Track designation, the FDA recognizes the potential of INZ-701 in ABCC6 Deficiency. We plan to work closely with the agency to establish an efficient path to approval. Receipt of Fast Track designation underscores our belief that INZ-701 could serve as an important therapy for patients living with ABCC6 Deficiency, notably for pediatric patients in whom this condition increases the risk of major clinical events such as stroke and severe neurological and cardiovascular disease,” said Douglas A. Treco, PhD, CEO, and chairman of Inozyme Pharma. “We look forward to presenting our development plans to regulatory agencies and reaching agreement on a pivotal study in pediatric patients with ABCC6 Deficiency by year-end 2024.”

The ongoing trial enrolled 10 patients with severe disease burden, evidenced by serious cardiovascular and retinal disease. Patients were divided into three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4) for 32 days. These doses were selected based on preclinical studies and PK/PD modeling. The primary endpoint of the study was the safety and tolerability of INZ-701 in adult patients with ABCC6 Deficiency.

Results showed a reduction in or stabilization of carotid intima-media thickness (cIMT), a marker for cardiovascular disease and stroke, and an increase in choroidal thickness, indicating potential benefits in vascular and retinal health. Additionally, four patients showed an improvement in visual function scores (VFQ-25), and nine patients experienced an improved global impression of change (GIC) score. PK and PD data revealed a sustained increase in plasma pyrophosphate (PPi) levels at the 1.8 mg/kg dose.

Deficiency

INZ-701 for ABCC6 Deficiency Shows No Severe Adverse Events, Demonstrates Good Tolerability

No severe adverse events (SAEs) were found in patients treated with INZ-701, with the medication being generally well tolerated. Minor adverse events included discoloration, discomfort, erythema, induration, pain, pruritus, warmth, fatigue, night sweats, and urticaria.

“We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” Treco stated. “Our investigations into the natural history of this disease have identified a substantial and previously overlooked pediatric population with a high risk of stroke. We believe these patients represent a critical unmet need in this genetic disease and that changes observed in adults treated with INZ-701 will translate to clinical benefits in a future trial in children.”

Currently, there are no therapies approved to treat ABCC6 Deficiency. This condition is caused by mutations in the ABCC6 gene, resulting in a reduction of systemic adenosine triphosphate (ATP) levels and low levels of pyrophosphate (PPi) and adenosine in the blood.

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“The high risk of ischemic stroke in pediatric patients with ABCC6 Deficiency and its devastating consequences represents a serious unmet need in this population,” commented Professor Zulf Mughal, MD, a consultant in pediatric bone disorders at Al Jalila Children’s Specialty Hospital in Dubai, UAE. “I am very encouraged to see that INZ-701 may improve vascular pathology and believe that this effect may translate to clinical benefits in patients of all ages.”

The Fast Track Designation by the FDA is a significant milestone for Inozyme Pharma and their ongoing efforts to develop INZ-701 as a viable treatment for ABCC6 Deficiency. With this designation, Inozyme can now benefit from more frequent interactions with the FDA, potential priority review, and a streamlined approval process, ultimately aiming to bring this much-needed therapy to patients as quickly as possible. The ongoing collaboration with the FDA and the planned pivotal study in pediatric patients highlight the commitment to addressing the critical needs of those affected by this genetic condition.

 

Resource: Inozyme, July 02, 2024

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