In a groundbreaking achievement, WuXi Biologics, a powerhouse in the world of Contract Research, Development, and Manufacturing Organizations (CRDMOs), extends its heartfelt congratulations to its strategic ally, Amicus Therapeutics.
The reason for celebration? None other than the U.S. FDA’s resounding approval for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules, a transformative two-component therapy.
This medical marvel is a lifeline for adults grappling with late-onset Pompe disease (LOPD), especially those who weigh ≥40 kg and have found little solace in their current enzyme replacement therapy (ERT).
Pombiliti™, a recombinant human GAA enzyme (rhGAA) fortified with high levels of bis-M6P (Mannose 6-Phosphate), is a testament to innovation that began as a mere concept back in 2012.
Now, it stands as a symbol of progress, brought to life by WuXi Biologics’ proprietary integrated technology platform and its unrivaled manufacturing prowess.
As the exclusive commercial drug substance manufacturer and a pivotal player in the supply chain of commercial drug products, WuXi Biologics stands tall.
They are the driving force behind the realization of Pombiliti™, with manufacturing services spanning five facilities across their extensive global network. The story is not just about a drug; it’s a testament to human ingenuity and the relentless pursuit of medical excellence.
Bradley Campbell, President and Chief Executive Officer of Amicus
In a triumph that reverberates through the corridors of the medical world, the approval of Pombiliti™ + Opfolda™ is more than just a milestone; it’s a beacon of hope for the Pompe disease community in the United States. This achievement is a testament to unwavering dedication and the formidable partnership forged between the WuXi Biologics team and Amicus.
Their shared commitment to patients and a relentless pursuit of excellence have given birth to a groundbreaking treatment option for those battling late-onset Pompe disease.
The journey from concept to molecule, from clinical manufacturing to commercial supply, has been a saga of perseverance and a testament to their unwavering promise to people affected by this rare disease.
It’s not merely a medicine; it’s a manifestation of years of hard work and an unyielding commitment to making a difference in the lives of those who need it most.
Dr. Chris Chen, the visionary CEO of WuXi Biologics, reflects on this momentous achievement with deep honor. It’s been over 11 years in the making, a journey filled with determination and the unwavering belief in their strategy – “follow the molecule.”
This victory not only reaffirms their path but also ignites the spark to accelerate the discovery, development, and manufacturing of more rare disease biologic therapies. It’s a testament to the power of collaboration and a shared passion for patients, a journey that promises to bring greater successes and benefits to patients worldwide battling rare diseases.
About Pompe Disease
Pompe disease, a formidable foe rooted in our genetic makeup, springs from an intricate puzzle involving a missing piece – the enzyme acid alpha-glucosidase (GAA). When this vital enzyme is in short supply or absent altogether, the consequences are dire.
Cells bear the burden as glycogen accumulates within, triggering the harbingers of Pompe disease. This condition spans a spectrum, from the heart-wrenching infantile form, where rapid decline casts a shadow over heart function, to the more gradual onset experienced in late-onset Pompe disease.
In this relentless battle, it’s not just skeletal muscles that face the brunt; the insidious grip extends to respiratory muscles, making each breath a challenging endeavor.
Late-onset Pompe disease is a formidable adversary, casting a shadow of progressive muscle weakness that engulfs the body, leaving no stone unturned, no muscle untouched, in its relentless march through time.
About Pombiliti + Opfolda
Pombiliti + Opfolda isn’t just your average therapy; it’s a dynamic duo, a two-part symphony in the world of medical innovation. Picture this: cipaglucosidase alfa-atga, a marvel of science, enriched with bis-M6P, reigns supreme as a recombinant human GAA (rhGAA).
It’s a master of high-affinity uptake through the M6P receptor, with an extra trick up its sleeve – the ability to morph into the most potent form of the enzyme.
But this journey wouldn’t be complete without its steadfast partner, miglustat, the oral enzyme stabilizer. Its mission? To stand guard against the loss of enzyme activity in the bloodstream, ensuring that the therapeutic dance remains in perfect harmony. Together, they are the dynamic duo, the dream team, rewriting the narrative for those battling Pompe disease.
About WuXi Biologics
In the realm of biologics, where science meets the art of healing, one name stands tall – WuXi Biologics. Think of them as the architects of hope, the wizards of discovery, development, and manufacturing, all rolled into one. Their mission? To unravel the mysteries of biologics, from inception to global impact, all in the name of patients worldwide.
With a formidable army of over 12,000 skilled minds spread across China, the United States, Ireland, Germany, and Singapore, WuXi Biologics doesn’t just provide solutions; they orchestrate miracles.
Armed with cutting-edge technology and boundless expertise, they are the architects of efficiency and cost-effectiveness in the world of biologics. As of June 30, 2023, they’re the backbone of 621 integrated client projects, with an impressive 22 in the throes of commercial manufacturing.
But WuXi Biologics is more than just a biotech juggernaut; they’re pioneers in embracing the Environmental, Social, and Governance (ESG) responsibilities. For them, it’s not just a checkbox; it’s ingrained in their very DNA and business strategy. Their facilities are the epitome of the future, harnessing next-generation biomanufacturing technologies and clean-energy sources.
To ensure their commitment to sustainability is unwavering, they’ve even set up an ESG committee, led by their visionary CEO, steering the ship toward a brighter, more responsible future. WuXi Biologics isn’t just changing the game; they’re rewriting the rules of what’s possible in the biologics CRDMO sector.