From the intricate realms of gene therapy to the precise targeting of antibody-drug conjugates, the past decade in medicine has been a thrilling voyage of discovery. Today, we embark on an enthralling journey through the ten most transformative drug discoveries of recent years, each one a beacon illuminating the future of therapeutics.
1. Palforzia (AR101) – Aimmune Therapeutics
Bringing a ray of hope to millions living with severe food allergies, Palforzia has inaugurated a new era in immunotherapy. It’s the world’s first FDA-approved treatment for peanut allergies, using oral immunotherapy to gradually desensitize individuals to peanut proteins. This game-changing approach is priced at approximately $890 per month, a small price to pay for freedom from a life-threatening allergy.
2. Zolgensma (onasemnogene abeparvovec-xioi) – Novartis
Coming in at an eye-watering $2.1 million for a one-time treatment, Zolgensma is currently one of the world’s most expensive drugs. But it’s also a revolution in gene therapy, offering a functional gene to combat spinal muscular atrophy, a leading genetic cause of infant mortality. This novel therapy has reshaped the landscape of a formerly devastating prognosis.
3. Prevymis (letermovir) – Merck & Co.
Prevymis, a first-in-class antiviral, marks a substantial leap in the fight against cytomegalovirus infection. With its novel mechanism of action—disrupting the viral terminase complex—it has carved out a new path in antiviral research. This pioneering drug is estimated to cost around $27,500 for a course of treatment, reflecting the value of its unique approach to tackling viral infections.
4. Luxturna (voretigene neparvovec) – Spark Therapeutics
Priced at around $850,000 for both eyes, Luxturna is a shining beacon in the field of gene therapy. As the pioneer of FDA-approved gene therapies for inherited diseases, Luxturna restores vision for patients suffering from a rare form of blindness caused by an RPE65 gene mutation. It introduces a normal copy of this gene into retinal cells, restoring the patient’s ability to convert light into electrical signals within the retina.
5. Risdiplam (Evrysdi) – Roche
Risdiplam, a novel therapy for spinal muscular atrophy, has made treatment more accessible with its oral administration. It enhances the production of the full-length survival motor neuron protein, bringing renewed hope to patients with this debilitating condition. The price of this life-altering therapy is approximately $340,000 per year.
6. Trikafta (elexacaftor/tezacaftor/ivacaftor) – Vertex Pharmaceuticals
Trikafta, a triumph in precision medicine priced at around $311,000 per year, has revamped the landscape of cystic fibrosis treatment. It targets the defective CFTR protein at multiple stages, drastically improving lung function and quality of life for up to 90% of cystic fibrosis patients.
7. Polivy (polatuzumab vedotin-piiq) – Genentech
Priced at around $165,000 for a course of treatment, Polivy, an antibody-drug conjugate, has revolutionized treatment for diffuse large B-cell lymphoma. It specifically targets CD79b, a protein prevalent in most B-cell malignancies, and delivers a potent cytotoxic agent to cancer cells.
8. Padcev (enfortumab vedotin-ejfv) – Astellas and Seattle Genetics
Another champion in the field of antibody-drug conjugates, Padcev targets Nectin-4, a protein highly expressed in urothelial cancers. By delivering a potent anti-cancer drug to these cells, it provides a targeted and effective therapeutic option for patients with advanced urothelial cancer. It comes with a price tag of approximately $110,000 to $120,000 for an average course of treatment.
9. Aduhelm (aducanumab) – Biogen
Coming in at an estimated cost of $56,000 per year, Aduhelm is the first new Alzheimer’s disease treatment in nearly two decades. As a monoclonal antibody, it targets the characteristic amyloid-beta plaques in the brain associated with Alzheimer’s disease. While its effectiveness has ignited discussions, Aduhelm represents a remarkable stride in our understanding and management of these challenging diseases.
10. CRISPR Therapies – Multiple companies
The development and application of CRISPR technologies have arguably been among the most significant breakthroughs in medicine in the last decade. This revolutionary gene-editing technology promises to treat a wide array of genetic diseases with unprecedented precision. The first CRISPR-based therapy, CTX001, has already been used to treat patients with beta-thalassemia and sickle cell disease. Although the exact price of CRISPR therapies may vary greatly, the first CRISPR-based treatment for a rare form of blindness was priced at $425,000 per eye, indicating the significant investment needed for these groundbreaking treatments.
The past decade, rich with transformative drug discoveries, has given new hope to patients and injected fresh vigor into the world of pharmacology. These milestones are not merely points of reflection but also beacons guiding future research and drug development. As we press forward, unwrapping the intricacies of human biology and disease, the next decade promises to be replete with even more exciting breakthroughs.
