2021-08-04| Trials & Approvals

After FDA Lifts Hold, Novartis to Proceed with Clinical Trials for Expensive Rare Disease Therapy

by Daniel Ojeda
Share To

Novartis’ Zolgensma (OAV-101) is a one-time gene therapy that uses an adeno-associated viral (AAV) vector to deliver a copy of the functional SMN gene to treat spinal muscular atrophy (SMA). To date, Zolgensma is approved in 41 countries, and more than 1,400 patients have been treated. With a price tag of more than $2 million, it is the most expensive gene therapy in the market. In 2020, Zolgensma raked in almost $1 billion in sales for Novartis.

In 2019, Zolgensma became the first FDA-approved gene therapy to treat children younger than 2 years old suffering from SMA. Soon after, it became embroiled in a scandal after reports of data manipulation involving top executives of AveXis, the company that initially developed the drug, emerged.

Later that year, Novartis suffered another major setback after the FDA imposed a partial clinical hold for trials for OAV-101 delivered into the spinal canal. However, that did not affect the marketing of Zolgensma or trials using an intravenous injection. The hold was imposed after an animal study showed inflammation of the dorsal root ganglia (DRG) mononuclear cell, resulting in neuronal damage.


FDA Lifts Clinical Hold

After almost 2 years, on August 3rd, Novartis announced that the FDA had lifted the partial hold following a comprehensive study conducted in non-human primates, which addressed the questions regarding the injury to dorsal root ganglia.

After consulting with the FDA and European Medicines Agency (EMA), Novartis is now moving forward with STEER, a global, randomized, double-blind, sham-controlled Phase 3 study to evaluate the safety, efficacy, and tolerability of OAV-101 delivered into the spinal canal. The trial will enroll treatment naïve patients with Type 2 SMA between the ages of 2 and 18 years of age who are able to sit but never walked. Significant unmet needs remain for these patients who suffer from a later-onset SMA.

“We are very pleased that our comprehensive nonclinical data package has addressed all issues identified related to DRG toxicity, and the FDA has reached the decision that we may proceed with our OAV-101 IT clinical trial program and initiate the STEER trial,” said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies.

“We believe that all patients diagnosed with SMA should be able to benefit from the transformative impact of gene therapy, and we remain confident that investigational OAV-101 IT is a viable potential treatment path for older patients who often have ongoing unmet needs, and for whom a one-time treatment could be especially compelling.”

This study will complement topline results from two Phase 3 clinical trials showing the efficacy of Zolgensma in patients younger than 6 weeks of age and pediatric patients presenting symptoms prior to treatment.

This news provides further validation for AAV-based genetic therapies, including therapies for Rett syndrome and Friedreich’s ataxia that are part of the Novartis pipeline.

© All rights reserved. Collaborate with us:
Related Post
Revolutionizing Treatment: ASGCT’s Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory Diseases
Advancing Health: ASGCT’s Clinical Trials Spotlight on Cell Therapy and Cell-Based Gene Therapy
Orchard Therapeutics’ Gene Therapy for Rare Neurological Disorder Wins FDA Approval
New Regulatory Measures in China A First in the Fight Against Sedentary Behavior in Children
Cure Genetics Announced Promising Safety and Efficacy Data of CAR-NKT Product CGC729 for RCC at ASGCT 2024
AstraZeneca’s $1.5 Billion ADC Manufacturing Facility in Singapore
Hims & Hers Health Inc. Tipping the Scales of the Weight Loss Market — and Wall Street is Loving it!
GV Announces Cooperation with CICC
Eli Lilly and Aktis Oncology Partner to Advance Novel Radiopharmaceuticals
Preventive Medicine for Neurodegeneration on the Rise: New Breakthrough in Routine Blood Testing for Alzheimer’s Disease
Scroll to Top