AstraZeneca Shares Climb on FDA Nod for Rare Disease Drug Koselugo
The regulator approved an expanded use for the drug to treat adults with a rare genetic disorder, bolstering the company's lucrative rare disease portfolio.
AstraZeneca Plc (NASDAQ: AZN) shares advanced toward a 52-week high in Wednesday trading after the U.S. Food and Drug Administration (FDA) approved an expanded adult indication for its rare disease drug, Koselugo.
The stock rose 0.63% to $89.55, pushing the pharmaceutical giant’s market capitalization to nearly $278 billion. The move reflects investor optimism as the company continues to strengthen its high-margin specialty drug pipeline.
The FDA greenlit Koselugo (selumetinib) for the treatment of adults with neurofibromatosis type 1 (NF1), a debilitating rare genetic condition, who have symptomatic, inoperable plexiform neurofibromas (PNs)—tumors that grow on nerve sheaths. This decision builds upon the drug's previous approval for pediatric patients, significantly widening its commercial potential.
According to the official FDA announcement, NF1 can cause a range of serious issues, including disfigurement, pain, and motor dysfunction. The approval offers a much-needed therapeutic option for adult patients who previously had limited choices for managing these complex tumors.
The regulatory decision was supported by robust data from the KOMET Phase 3 trial. The study demonstrated a confirmed overall response rate—a measure of tumor shrinkage—of 20% in patients receiving Koselugo, compared to just 5% in the placebo group by the 16th cycle of treatment. The safety profile in adults was consistent with that observed in earlier pediatric studies.
This latest approval is a strategic victory for AstraZeneca, reinforcing the value of its $39 billion acquisition of Alexion Pharmaceuticals in 2021, which established the company as a major force in rare diseases. By expanding Koselugo's label, AstraZeneca is better positioned to maximize revenue from the drug and solidify its presence in a market characterized by high unmet need and significant pricing power.
The positive news adds to a month of upward momentum for AstraZeneca. The company’s stock has been on a steady climb, supported by a stream of positive data from its oncology portfolio presented at the European Society for Medical Oncology (ESMO) Congress in October. This dual strength in both oncology and rare diseases has been a key driver of investor confidence.
Wall Street sentiment remains broadly positive, with a consensus 'Strong Buy' rating from analysts and an average price target of approximately $93.15. The Koselugo approval provides further fundamental support for this bullish outlook, demonstrating the company's ability to successfully navigate the regulatory process and bring innovative treatments to market.
Looking forward, investors will be watching the commercial rollout of the expanded indication and its contribution to the bottom line of AstraZeneca's Rare Disease division. The success of Koselugo in this new adult population will serve as a key indicator of the company’s ongoing strategy to diversify its revenue streams beyond its blockbuster cancer drugs.