together with Jen Huen, Naveed Yasin, Becca Burnham, Arathi Chintireddy, and Adrian Parodi
This deep dive into 2021’s novel large molecule drug approvals is the 2nd in a series of focus articles on 2021’s novel large molecule drugs. See part one here.
For each of the 16 large molecule drug approvals of 2021 (excluding vaccines), we’ll explore:
- how they work (MoA)
- key approval endpoints and disease context
- how they’re different from previous treatments
- how they were discovered
- and more scientific highlights.
One example from the 16 slides is avalglucosidase alfa-ngpt (Nexviazyme), Sanofi Genzyme’s second generation GAA replacement therapy:
Nexviazyme (avalglucosidase alfa) is Sanofi Genzyme’s second-generation GAA (acid alpha-glucosidase) replacement therapy, after Lumizyme (alglucosidase alfa). Both drugs are used to treat glycogen storage disease type II (Pompe disease), a rare lysosomal storage disorder caused by mutations in GAA. Glycogen accumulation in the lysosomes of muscle cells results in progressive muscle weakness, impaired motor function, and respiratory decline that leads to mortality.
The molecule’s glycosylation state was found to be key to its stability in blood and for muscle cell uptake. The molecule was engineered to bear more mannose 6-phosphates compared to Lumizyme, resulting in better uptake and glycogen clearance.
For site-wide searchability, all summaries appear below to premium members, but it’s much easier to flip through your premium slide deck 😉. You can find the poster in part one and the small molecules series here).
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