Prix Galien Awards 2020 Announced: Multiple 'First-in-Class' Innovations Honored

Today, the Galien Foundation announced the winners of the 2020 Prix Galien Awards. Regarded as the “Nobel Prize of the pharmaceutical industry,” the Prix Galien is widely recognized as the highest honor in the pharmaceutical and biotechnology sectors, celebrating outstanding scientific innovations that improve human health. This year marks the 50th anniversary of the award; since its inception in 1970, 268 biopharmaceutical products have been honored across 18 therapeutic areas.

Image source: Galen Foundation official website

The Galien Awards are selected by a jury comprising multiple Nobel laureates. The awards include the Best Pharmaceutical Product, the Best Biotechnology Product, and the Best Medical Technology. All awarded products must have received FDA approval for market launch within the past five years and demonstrate the potential to significantly impact human health. This year, the Galien Foundation also introduced the Best Digital Health Product award for the first time, recognizing the indispensable role of digital health solutions in healthcare systems. This year’s winners are:

Best Medicine Award: Pretomanid, Global Alliance for TB Drug Development (TB Alliance)

Pretomanid is a new molecular entity developed by the non-profit organization Global Alliance for TB Drug Development (TB Alliance). It is used in combination with bedaquiline and linezolid to treat patients with specific forms of highly drug-resistant tuberculosis (TB). Approved by the FDA in 2019, it is the third new anti-tuberculosis drug approved by the FDA in nearly 40 years and the first new tuberculosis drug developed and marketed by a non-profit organization.

Due to the increasing prevalence of bacterial resistance, multidrug-resistant TB (MDR-TB) and extensively drug-resistant TB (XDR-TB) have become significant threats to human health. According to World Health Organization (WHO) statistics, there were 490,000 new cases of MDR-TB worldwide in 2016, a small proportion of which were XDR-TB. These patients often exhibit intolerance or develop resistance to multiple therapies, resulting in very limited treatment options. Currently, most patients with XDR-TB may require treatment with up to eight antibiotics for a duration of 18 months or longer. The WHO estimates that the treatment success rate for patients with XDR-TB is approximately 34%.

▲Molecular structure of pretomanid (Image source: Fvasconcellos 18:28, 3 June 2007 (UTC) [Public domain])

In a pivotal clinical trial, 109 patients with extensively drug-resistant tuberculosis (XDR-TB) and multidrug-resistant tuberculosis (MDR-TB) who were intolerant to or unresponsive to existing therapies received a combination regimen consisting of pretomanid, bedaquiline, and linezolid. The trial results demonstrated that after six months of treatment, the success rate of this combination therapy reached 89%, significantly higher than the historical success rates for treating XDR-TB patients.

Best Medical Technology Award: MitraClip Transcatheter Mitral Valve Repair System, Abbott

MitraClip is the world’s first transcatheter mitral valve repair (TMVr) treatment system, offering a therapeutic option for certain patients with primary or secondary mitral regurgitation who would otherwise be ineligible for treatment. MitraClip has been used to treat more than 80,000 patients worldwide.

Image source: Abbott official website

Best Biotechnology Product Award:

Tegsedi, Akcea Therapeutics, and Ionis Pharmaceuticals

Onpattro, Alnylam Pharmaceuticals

Vyndaqel，Pfizer

Hereditary Transthyretin-Mediated Amyloidosis (hATTR) is a fatal, progressive genetic disorder caused by the misfolding of transthyretin (TTR) protein in patients, leading to the deposition of amyloid TTR in various tissues and organs throughout the body. TTR deposits occur in tissues including peripheral nerves, heart, gastrointestinal tract, eyes, kidneys, central nervous system, thyroid, and bone marrow. The accumulation of TTR in these tissues and organs results in multiple sensory, motor, and autonomic dysfunctions, ultimately leading to patient death within 3–5 years after symptom onset. It is estimated that there are approximately 50,000 hATTR patients worldwide.

This year, three co-winning products address this disease from different perspectives. Tegsedi is an antisense oligonucleotide drug that inhibits human TTR synthesis. By binding to the mRNA encoding the TTR protein, it induces mRNA degradation, thereby reducing levels of TTR protein (both wild-type and mutant). It is administered as a once-weekly subcutaneous injection, allowing patients to self-administer at home.

Onpattro, developed by Alnylam, is an RNAi therapy targeting transthyretin (TTR). This class of drugs works by silencing specific mRNA molecules involved in disease pathogenesis. Onpattro encapsulates siRNA within lipid nanoparticles, delivering the drug directly to the liver via infusion. By binding to the mRNA encoding abnormal TTR, it inhibits TTR production, thereby helping to reduce the accumulation of amyloid deposits in peripheral nerves, alleviate symptoms, and assist patients in better managing their condition. It is also the first RNAi therapy approved by the FDA.

Vyndaqel, developed by Pfizer, is an oral TTR stabilizer that binds specifically to TTR, stabilizing its tetrameric structure and thereby delaying the formation of amyloid deposits. It has been approved by the FDA for the treatment of cardiomyopathy caused by wild-type or hereditary transthyretin-mediated amyloidosis.

Best Digital Health Product: Somryst, Pear Therapeutics

Somryst, developed by Pear Therapeutics, is the first prescription digital therapeutic (PDT) approved by the FDA for the treatment of insomnia. Somryst is designed to provide personalized neurobehavioral interventions, primarily utilizing algorithm-driven cognitive behavioral therapy for insomnia (CBT-I), to improve insomnia symptoms and offer patients a first-line treatment.

In addition, Somryst provides a clinician dashboard that allows healthcare providers to track patient treatment and disease progression. The clinician dashboard can display information on patients’ use of Somryst, including the Insomnia Severity Index (ISI), the Patient Health Questionnaire (PHQ-8), and sleep metrics derived from nightly sleep diaries.

References:

[1] The Galien Foundation. Retrieved October 29, 2020, from https://www.galienfoundation.org/

[2] The Galien Foundation Introduces 2020 Prix Galien USA Nominees in "Best Biotechnology Product," "Best Pharmaceutical Agent," and "Best Medical Technology" Categories. Retrieved October 29, 2020, from https://www.prnewswire.com/news-releases/the-galien-foundation-introduces-2020-prix-galien-usa-nominees-in-best-biotechnology-product-best-pharmaceutical-agent-and-best-medical-technology-categories-301099587.html?tc=eml_cleartime

[3] The Galien Foundation Debuts 2020 Prix Galien Nominees in New Category for "Best Digital Health Product". Retrieved October 29, 2020, from

https://www.prnewswire.com/news-releases/the-galien-foundation-debuts-2020-prix-galien-nominees-in-new-category-for-best-digital-health-product-301143986.html

[4] MitraClip. Retrieved October 29, 2020, from https://www.cardiovascular.abbott/us/en/hcp/products/structural-heart/mitraclip.html

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