Median Drug Prices For Rare Diseases Hit $300K In 2023, Report Highlights Lack of Clear Rationale Behind Escalating Drug Prices
Portfolio Pulse from Vandana Singh
In 2023, the median annual list price for new drugs in the U.S. reached $300,000, a 35% increase from 2022, with a focus on rare diseases. Regeneron's Veopoz was the most expensive at $1.8 million annually. Gene therapies ranged from $2.2 million to $3.2 million. Over half of the FDA-approved drugs targeted orphan diseases. Concerns were raised about the lack of clear rationale behind these escalating prices.

February 26, 2024 | 5:14 pm
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CRISPR Therapeutics, partnering with Vertex Pharmaceuticals, launched Casgevy for sickle cell disease at $2.2 million, marking a significant entry into high-cost gene therapies.
CRISPR Therapeutics' collaboration with Vertex on the high-priced Casgevy gene therapy could lead to a positive short-term impact on its stock, highlighting its role in pioneering expensive, innovative treatments.
CONFIDENCE 80
IMPORTANCE 75
RELEVANCE 80
POSITIVE IMPACT
Regeneron's Veopoz is the most expensive drug for consistent use in 2023, priced at $1.8 million annually for treating CHAPLE disease.
Veopoz's high price point and its mention as the most expensive drug could attract investor interest in Regeneron, potentially boosting its stock in the short term due to the visibility and the premium pricing of its product.
CONFIDENCE 85
IMPORTANCE 80
RELEVANCE 90
POSITIVE IMPACT
Sarepta Therapeutics priced its gene therapy for muscular dystrophy, Elevydis, at $3.2 million, making it one of the most expensive gene therapies in 2023.
Sarepta Therapeutics' Elevydis, priced at $3.2 million, could see a positive short-term stock impact due to its high price and the attention it brings to the company's innovative approach to treating muscular dystrophy.
CONFIDENCE 85
IMPORTANCE 80
RELEVANCE 85
POSITIVE IMPACT
Vertex Pharmaceuticals, in collaboration with CRISPR Therapeutics, priced their gene therapy for sickle cell disease, Casgevy, at $2.2 million.
The high pricing of Casgevy, a gene therapy for sickle cell disease, could positively impact Vertex Pharmaceuticals' stock in the short term due to the innovative nature of the treatment and its significant price point.
CONFIDENCE 80
IMPORTANCE 75
RELEVANCE 80