New $4.25M Drug Hits the Market, Signaling Big Pharma Trends – The Average Joe
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    New $4.25M Drug Hits the Market, Signaling Big Pharma Trends


    March 23, 2024

    Living can be expensive… but you probably already know that from your grocery bills. For the rare few born with metachromatic leukodystrophy (MLD), surviving means shelling out a staggering $4.25M. Last week, the FDA gave the green light to LENMELDY, a groundbreaking gene therapy from Kyowa Kirin’s ($KYKOF) Orchard Therapeutics — making it the most expensive drug in the world.

    • MLD affects ~1 in every 100K live births, disrupts most functions, including speech and mobility, and is often fatal within five years.
    • Orchard justifies the eye-popping price tag by asserting it “reflects its clinical, economic and societal value” (BBG).

    Nature or nurture? The answer is both. Gene therapies, which modify the genome of specific cells, have made significant progress in recent years. Bloomberg predicts annual gene therapy spending could skyrocket from $5.15B in 2020 to $25.3B in 2026. The number of gene therapies in development surged from just under 200 in 2011 to a staggering 1,745 in 2021 — predominantly targeting rare diseases and commanding million-dollar price tags.

    Read: Vertex ($VRTX) made a fortune pushing a rare disease into extinction, but now it’s betting its future on gene therapies.

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