Pompe Disease Treatment Market: By Treatment Type (Enzyme Replacement Therapy, Gene Therapy), By Age Group Type (Infantile-onset Pompe Disease, Late-Onset Pompe Disease), and Geography    

Pompe Disease Drug Sales was valued at USD 1.2 billion in 2022 and projected to reach USD 1.8 billion by 2027, at a CAGR of 8% from 2024-2030. Pompe illness is a genetic metabolic ailment that affects babies and is caused by mutations in the GAA gene, which produces the acid alpha-glucosidase enzyme, which reduces glycogen to a simple form. The lack or mutation of the GAA gene causes glycogen buildup, which causes heart problems, muscle weakness, and liver damage, which can lead to early death.

How will Maze Therapeutics deal benefit the Sanofi?

• May 1st, 2023, Sanofi made a deal with Maze for the MZE001 that treats Pompe patients.  As part of the agreement, Maze will receive USD150 Million in Upfront Cash and Equity Investment, as well as USD 600 Million upon reaching various development, regulatory, and commercial milestones. This exclusive deal provides Sanofi with access to Maze's GYS1-targeting initiatives and intellectual property.
• Maze (MZE001) basically works on the glycogen synthase (GYS1) inhibitor which is responsible for the glycogen production. Inhibition of this enzyme will play a role in the glycogen production process. Based on the phase 1 clinical study, MZE001 480 mg BID x 10 days safely and substantially reduced acute glycogen synthesis and lowered total muscle glycogen in healthy adults. Combining the ERT + Glycogen Synthase Inhibitor will significantly improve the glycogen breakdown.
• Maze’s deal with Sanofi also gives the French pharmaceutical company exclusive rights to related GYS1-targeting programs and intellectual property. Maze could receive up to $600 million more if certain development, commercial and regulatory milestones are met.
• An interesting aspect of this collaboration is that it will give Sanofi an edge in the Pompe Disease space where it will face stiff competition from Amicus, a two-component therapy which recently received approval from the CHMP. By partnering with Maze, Sanofi is better positioned to gain ground in this Pompe Disease market.

Pompe Disease Treatment Market Key Developments:

• In April 2023, Amicus Therapeutics received positive CHMP opinion for Opfolda (miglustat) used for the treatment of Pompe Disease
• In March 2023, Amicus Therapeutics received European Commission approval for a enzyme replacement therapy, Pombiliti (cipaglucosidase alfa) in combination with Opfolda for adults with late-onset Pompe disease (LOPD)
• In March 2023, Sanofi Aventis Korea received Ministry of Food and Drug Safety approval for the Nexviazyme (Avalglucosidase Alfa-ngpt) for the treatment of pome disease
• In January 2023, FDA lifted the hold on Astellas Pharma’s FORTIS Ph1/2 clinical trial for gene therapy, AT 845 for the treatment of Pompe Disease