The battle for dominance in the Pompe disease treatment market begins in earnest … Sanofie’s Amicus challenge

[헬스코리아뉴스 / 이충만] Competition is fierce in the global market for the treatment of Pompe disease.

Pompe disease is a rare disease in which muscles gradually weaken. Due to the deficiency of alpha-glucosidase (GAA) enzyme, glycogen complex sugar accumulates in skeletal muscle and myocardium, leading to muscle weakness, respiratory failure, and premature death due to heart failure. The current standard of care is enzyme replacement therapy (ERT).

France’s Sanofi is regarded as a traditional powerhouse in the Pompe disease treatment market. Sanofi’s enzyme replacement therapy ‘Myozyme’ was approved by the US Food and Drug Administration (FDA) in 2006 for the first treatment for Pompe disease. there is a bar

The competitor is American Amicus Therapeutics. In 2013, the company acquired Callidus Biopharma of the United States and secured a candidate material for enzyme replacement therapy for Pompe disease, quickly emerging as a competitor to Sanofi.

After that, the two companies started developing a new treatment for Pompe disease to secure market dominance. Competing drugs are Sanofi’s ‘Nexviazyme’ (ingredient name: avalglucosidase, α-ngpt) and Amicus’ ‘AT-GAA’.

Among them, Sanofi’s ‘Nexviazyme’ received FDA approval last year as a treatment for Pompe disease, taking a step ahead of the competition. Amikus’ ‘AT-GAA’, which is currently under development, is also expected to be approved by the FDA within this year, so the battle for market leadership is expected to begin in earnest.

Sanofi’s Pompe disease treatment ‘Nexviazyme’ approved by FDA last year

Lumizyme [사진=사노피 홈페이지]
Lumizyme [사진=사노피 홈페이지]

Sanofi’s ‘Nexviazyme’ is an intravenous injection for the treatment of late-onset Pompe disease over 1 year old. The mechanism of ‘nexviazyme’ targets the M6P receptor, which is a major pathway for GAA enzyme to be absorbed into the cell’s lysosome.

This treatment is a follow-up formulation developed as the patent expired in the United States in 2018 for the existing ‘Lumizyme’. ‘Lumizyme’ is a blockbuster with annual sales of 1.11 billion dollars (about 1.4 trillion won) as of 2020, and it was essential for Sanofi to develop an alternative drug.

Sanofi conducted a phase 3 clinical trial (test name: COMET) in 100 patients with late-onset Pompe disease. As a result, the ‘Nexviazyme’ administration group showed positive results in the measures of respiratory function enhancement and walking distance.

Based on these results, Sanofi submitted a biopharmaceutical new drug license application (BLA) to the FDA, and the FDA approved it as a treatment for late-onset Pompe disease over 1 year old in August 2021.

According to Sanofi, in the study, the ‘Nexviazyme’ group increased the M6P receptor about 15 times compared to the existing treatment group, increasing cellular enzyme uptake and improved glycogen reduction in muscle tissue.

FDA to announce approval of Amikus ‘AT-GAA’ in October this year

Amicus Logo

Amicus’ ‘AT-GAA’ is a competitive drug that tried to enter the market with the aim of expiring the US patent for ‘Lumizyme’.

However, in February 2021, it was found that ‘AT-GAA’ did not meet the primary endpoint in a phase 3 clinical trial for patients with Pompe disease. At that time, the share price of Amicus fell 37% to $12.57 from the previous day ($20.09).

Amikus then re-analyzed the data, refining the details of the clinical data, and concluded that the results were “nominal and statistically significant.”

Based on these results, Amicus applied for a BLA for ‘AT-GAA’ with the FDA, and the FDA received it and set the date of announcement of approval under the Prescription Drug Applicant Fee Act (PDUFA) on August 29 of this year.

However, the FDA rescheduled the announcement on October 10 (local time) to October 29, 2022. It has been extended by 3 months more than before. In this regard, Amicus said, “The deadline has been extended to fully review the submitted information,” and “there was no request related to additional clinical data.”

On the same day, John F. Crowley, CEO of Amicus, expressed his aspiration that “‘AT-GAA’ will become a new standard treatment for Pompe disease.”

‘AT-GAA’ is an enzyme replacement therapy consisting of a recombinant alpha-glucosidase (rhGAA) enzyme and ‘ATB200’, a type of M6P. The FDA designated this treatment as the first innovative drug for Pompe disease in 2019.

Separately, the Drug User Advisory Committee (CHMP) under the European Medicines Agency (EMA) received an application for approval of ‘AT-GAA’ last year, and it is known that approval will be decided at the end of this year.

Amicus Therapeutics is an American biotechnology company that develops treatments for rare diseases. On the 10th (local time), Amicus on the Nasdaq closed higher at $6.74, up 8.19% from the previous day’s closing price of $6.23.

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