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Novartis To Launch New Phase 3 Study To Expand Use of Spinal Muscular Atrophy Drug

Zolgensma, originally approved by the FDA in 2019, is the world's most expensive medicine at $2.125 million per treatment.

Image source: Novartis

Swiss drugmaker Novartis AG (NYSE: NVS ) will launch a new phase three study to expand the use of Zolgensma — the world’s most expensive medicine — following the US Food and Drug Administration’s (FDA) decision to lift a halt on clinical trials

On Tuesday, Novartis said it plans to study the use of the gene therapy for for the treatment of spinal muscular atrophy (SMA) in patients between the ages of 2 and 18.

Approved by the FDA in May 2019 for children under the age of two, Zolgensma (onasemnogene abeparvovec-xioi) is a one-time infusion designed to delay or prevent disability altogether by replacing a faulty gene or restoring production of a key muscle protein.

The FDA charged Novartis in August 2019 with concealing manipulated data during the approval application process. The agency said the inaccurate data didn't affect the safety or efficacy of the drug and ultimately took no punitive action.

Additionally, regulators halted testing in October 2019 of a new formulation of Zolgensma for older patients after safety concerns stemming from an animal study.  

Novartis announced on Tuesday the FDA would allow existing trials to proceed following a review of newly-supplied data, thereby lifting the partial hold.

In a statement, Shephard Mpofu, Novartis Gene Therapies’ chief medical officer, said, “We are very pleased that our comprehensive nonclinical data package has addressed all issued identified related to DRG [dorsal root ganglia] toxicity and the FDA has reached the decision that we may proceed.”

“We believe that all patients diagnosed with SMA should be able to benefit from the transformative impact of gene therapy,” Mpofu said. 

He added that the company believes Zolgensma “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.”

The new trial will build upon previous findings that showed treatment with the therapy resulted in clinically meaningful response in patients between the ages of 2 and 5 years old.

SMA, a deadly disease caused by a genetic defect, weakens a person’s muscles so dramatically that they become unable to move and can eventually impair the ability to swallow or breathe. Symptoms typically start between 6 and 18 months of age. Without treatment, some forms of SMA can be fatal.

In Novartis’s most recent study, for which data was released in June, children with the degenerative disorder achieved several key milestones after treatment with the drug, including sitting unassisted and standing. 

More than 1,200 children globally have been treated with Zolgensma to date, including patients in the US, European Union, South Korea and Canada, according to Novartis.

Kenneth Hobby, president of non-profit organization Cure SMA, said the new trial “has the potential to open up access for older patients to all the benefits of gene therapy.”

“We have seen the interest among our symptomatic patients and their families in gene therapy and this study is an important step in understanding its potential to address unmet needs that remain in the SMA community,” Hobby said in a statement.

Sporting a listing price of $2.125 million, Zolgensma is the most expensive medicine in the world. 
Novartis, which has maintained that the drug’s nature as a one-time treatment and strong data support the extraordinary price, offers insurers the ability to pay $425,000 per year over a five-year span. 

At least 11 insurers in the US have policies in place to cover the cost of the treatment.


Source: Equities News

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