The field of genetic disease treatment is experiencing a golden era of sorts. In 2024, blockbuster therapies not only continued to deliver strong financial performance but also expanded access across broader patient populations. From RNA interference (RNAi) breakthroughs to next-generation triple combination modulators, discover the therapies set to redefine genetic disease treatment in 2025 and beyond.
When it comes to companies that report in foreign currencies, the conversion to US dollars uses the average annual exchange rates reported by the US Federal Reserve.
Related: Top 50 Best-Selling Drugs to Watch in 2025: Insights from 2024 Sales Data
1. Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor)
Indication(s): Cystic fibrosis
Manufacturer(s): Vertex Pharmaceuticals Inc.
2024 Sales (USD): $10.24 billion
2025 Outlook: Approved for cystic fibrosis patients two years of age and older with at least one F508del mutation or other responsive CFTR mutations, Trikafta generated $10.24 billion of Vertex’s $11.02 billion in product revenue in 2024, up 12% from 2023. The FDA expanded its label in December 2024 to include children as young as two with at least one F508del or other responsive CFTR mutations, significantly broadening its reach. Effective in about 90% of cystic fibrosis patients and shown to improve lung function and quality of life, Trikafta is now the standard of care in many countries. With approvals in over 60 markets and new access deals, including one with the UK’s NICE, its strong clinical profile and market growth position it as a key genetic therapy to watch in 2025.
2. Spinraza (nusinersen)
Indication(s): Spinal muscular atrophy (SMA)
Manufacturer(s): Ionis Pharmaceuticals and Biogen
2024 Sales (USD): $1.57 billion
2025 Outlook: Spinraza remains a genetic disease drug to watch in 2025 after generating around $1.6 billion in global sales in 2024, positioning it as one of the leading therapies in rare neuromuscular diseases. Developed through a collaboration between Ionis Pharmaceuticals and Biogen, Spinraza became the first FDA-approved treatment for SMA in 2016 and is administered via intrathecal injection. It’s approved for all types of SMA, from infantile-onset through later-onset forms, helping significantly improve motor function and survival outcomes in affected patients. Despite facing pressure from newer competitors and experiencing a slight sales decline, Biogen is advancing a higher-dose regimen (DEVOTE) with improved efficacy data and pending regulatory review, which could reinvigorate the franchise in 2025. Biogen’s intrathecal therapy for SMA marked a 13% decline to approximately $1.15 billion in global revenue during the first nine months of 2024. While eclipsed by newer SMA treatments, Spinraza’s enduring presence, especially where access to oral and gene therapies is limited, makes it a key player in the SMA landscape.
3. Takhzyro (lanadelumab‑flyo)
Indication(s): Routine prophylaxis to prevent attacks of hereditary angioedema (HAE)
Manufacturer(s): Takeda
2024 Sales (USD): $1.43 billion
2025 Outlook: Takhzyro is a genetic disease drug to watch in 2025, driven by its strong sales performance in 2024. Approved by the FDA and EMA, Takhzyro is indicated for routine prophylaxis to prevent attacks of HAE in patients aged two years and older. It is a fully human monoclonal antibody that inhibits plasma kallikrein, a key enzyme involved in the pathway that leads to HAE attacks. Takhzyro generated around $1.2 billion in revenue in fiscal year 2024 (ending March 31, 2024) and $1.4 billion in fiscal year 2025, ending March 31, 2025, representing an 18.9% year-over-year increase at constant exchange rate (CER). The increase in sales was driven by higher demand across the US, Europe and Canada. The drug remained the top sales generator in Takeda’s rare diseases segment, which saw a 4.6% year-over-year growth in 2025.
4. Crysvita (burosumab)
Indication(s): Treatment of X-linked hypophosphatemia (XLH), management of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO)
Manufacturer(s): Kyowa Kirin and Ultragenyx
2024 Sales (USD): $1.42 billion
2025 Outlook: Crysvita, a therapy jointly developed and commercialized by Ultragenyx Pharmaceutical Inc. and Kyowa Kirin, has established itself as a leading treatment for rare phosphate-wasting disorders. In 2024, Crysvita generated approximately $410 million in sales for Ultragenyx, which exceeded the company’s prior guidance, and over $1 billion for Kyowa Kirin. In its annual financial report for 2024, Kyowa Kirin reported continued growth of Crysvita in North America and EMEA, with market availability now expanded to 52 countries and regions. Kyowa said continued disease awareness activities, particularly in emerging markets like Latin America and Türkiye, and the enhancement of patient support programs in North America, including transitional care for Crysvita from pediatric to adult patients, along with expansion into additional indications such as TIO, are helping drive its continued growth. Analysts expect Crysvita to continue to grow at a robust pace, with long-term projections supported by increased patient access and continuing geographic expansion. For 2025, Ultragenyx projects full-year revenue for Crysvita in the range of $460 million to $480 million, representing growth of about 14% to 19% compared to 2024. The first quarter of 2025 delivered $103 million in Crysvita sales, followed by $120 million in the second quarter.
5. Amvuttra (vutrisiran)
Indication(s): Amvuttra is approved for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN). In March 2025, the US FDA granted approval for an expanded indication, transthyretin amyloid cardiomyopathy (ATTR-CM), making it the first RNAi therapy authorized for both neurological and cardiac forms of the disease.
Manufacturer(s): Alnylam Pharmaceuticals
2024 Sales (USD): $972 million
2025 Outlook: Amvuttra is an RNAi therapeutic developed and commercialized by Alnylam Pharmaceuticals, indicated for hATTR-PN. In March 2025, it won expanded approval in ATTR-CM, becoming the first RNAi drug approved for both neurological and cardiac forms of the disease. In 2024, it generated approximately $970 million in revenue. In 2025, Amvuttra is experiencing exceptional growth following its expanded FDA approval for ATTR-CM in March 2025. In the second quarter alone, Amvuttra delivered about $492 million in sales. On the strength of this performance, Alnylam raised its 2025 net product revenue projection to a range of $2.65 billion to $2.80 billion for its transthyretin (TTR) franchise guidance, which includes Amvuttra and Onpattro (patisiran). Analysts further project that Amvuttra sales could grow to as much as $5 billion by 2029, reflecting both the strong market uptake and its new indication in ATTR-CM.
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