We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
AZN Wins FDA Nod for Expanded Use of Rare Disease Drug, Koselugo
Read MoreHide Full Article
Key Takeaways
AstraZeneca gained FDA approval to extend Koselugo use to adult NF1 patients with symptomatic, inoperable PN.
Koselugo's label was also recently expanded in the EU, Japan and other countries, with more reviews ongoing.
AZN's KOMET study showed a 20% overall response rate for Koselugo versus 5% with placebo by cycle 16.
AstraZeneca (AZN - Free Report) announced that the FDA has approved its oral, selective MEK inhibitor, Koselugo (selumetinib), for expanded use. The drug is now indicated to treat symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1) in the United States.
Koselugo’s label was also recently expanded to include this indication in the EU, Japan, and some other countries, with additional regulatory reviews currently ongoing. Koselugo is already approved to treat certain pediatric patients with NF1 who have symptomatic, inoperable PN in the United States, the EU, Japan, China and other countries.
In the United States, a granule formulation of Koselugo was also recently approved by the FDA for young children aged one year and older with NF1 PN.
Rationale Behind the FDA Nod for AZN’s Koselugo Expanded Use
The FDA approval of the label expansion of AstraZeneca’s Koselugo to treat adult patients with NF1 who have symptomatic, inoperable PN was backed by data from the global phase III KOMET study. In the primary endpoint analysis of the KOMET study, treatment with Koselugo demonstrated a statistically significant and clinically meaningful overall response rate (ORR) of 20% compared to 5% with placebo by cycle 16.
ORR represents the proportion of patients who achieve either a confirmed complete response (tumor disappearance) or a partial response (a tumor volume reduction of at least 20%).
Additionally, approximately 86% of patients receiving Koselugo therapy achieved an observed duration of response of at least 6 months. Year to date, shares of AstraZeneca have rallied 35.3% compared with the industry’s growth of 15.8%.
Image Source: Zacks Investment Research
NF1 is a rare, progressive genetic disorder typically identified in early childhood but often advancing into adulthood, with the potential to affect multiple organ systems. Per AZN, up to half of individuals with NF1 develop non-cancerous PN in the brain, spinal cord, or nerves, which may emerge later in life and grow substantially, causing disfigurement, pain, and various functional impairments.
Koselugo enjoys the FDA’s Orphan Drug designation in the United States, the EU, Japan and other countries for the treatment of NF1.
AstraZeneca has a collaboration deal with Merck (MRK - Free Report) for Koselugo. In the third-quarter 2025 earnings release, AstraZeneca announced modifying the Koselugo partnership with Merck, having assumed full global responsibility for the drug’s costs, revenues and profits. Previously, AZN and MRK split Koselugo’s pre-tax profits and losses equally. Apart from Koselugo, AstraZeneca and Merck have a collaboration deal for Lynparza, which is ongoing.
In the past 60 days, estimates for Editas Medicine’s loss per share have narrowed from $2.12 to $2.06 for 2025. During the same time, loss per share estimates for 2026 have widened from $1.02 to $1.05. Year to date, shares of EDIT have rallied 94.5%.
Editas Medicine’searnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 13.17%.
In the past 60 days, estimates for ADMA Biologics’ earnings per share have increased from 57 cents to 58 cents for 2025. During the same time, earnings per share estimates for 2026 have improved from 88 cents to 90 cents. Year to date, shares of ADMA have lost 4.6%.
ADMA Biologics’ earnings beat estimates in one of the trailing four quarters, matched once, and missed the same on the remaining two occasions, with the average negative surprise being 3.01%.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
AZN Wins FDA Nod for Expanded Use of Rare Disease Drug, Koselugo
Key Takeaways
AstraZeneca (AZN - Free Report) announced that the FDA has approved its oral, selective MEK inhibitor, Koselugo (selumetinib), for expanded use. The drug is now indicated to treat symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1) in the United States.
Koselugo’s label was also recently expanded to include this indication in the EU, Japan, and some other countries, with additional regulatory reviews currently ongoing. Koselugo is already approved to treat certain pediatric patients with NF1 who have symptomatic, inoperable PN in the United States, the EU, Japan, China and other countries.
In the United States, a granule formulation of Koselugo was also recently approved by the FDA for young children aged one year and older with NF1 PN.
Rationale Behind the FDA Nod for AZN’s Koselugo Expanded Use
The FDA approval of the label expansion of AstraZeneca’s Koselugo to treat adult patients with NF1 who have symptomatic, inoperable PN was backed by data from the global phase III KOMET study. In the primary endpoint analysis of the KOMET study, treatment with Koselugo demonstrated a statistically significant and clinically meaningful overall response rate (ORR) of 20% compared to 5% with placebo by cycle 16.
ORR represents the proportion of patients who achieve either a confirmed complete response (tumor disappearance) or a partial response (a tumor volume reduction of at least 20%).
Additionally, approximately 86% of patients receiving Koselugo therapy achieved an observed duration of response of at least 6 months. Year to date, shares of AstraZeneca have rallied 35.3% compared with the industry’s growth of 15.8%.
Image Source: Zacks Investment Research
NF1 is a rare, progressive genetic disorder typically identified in early childhood but often advancing into adulthood, with the potential to affect multiple organ systems. Per AZN, up to half of individuals with NF1 develop non-cancerous PN in the brain, spinal cord, or nerves, which may emerge later in life and grow substantially, causing disfigurement, pain, and various functional impairments.
Koselugo enjoys the FDA’s Orphan Drug designation in the United States, the EU, Japan and other countries for the treatment of NF1.
AstraZeneca has a collaboration deal with Merck (MRK - Free Report) for Koselugo. In the third-quarter 2025 earnings release, AstraZeneca announced modifying the Koselugo partnership with Merck, having assumed full global responsibility for the drug’s costs, revenues and profits. Previously, AZN and MRK split Koselugo’s pre-tax profits and losses equally. Apart from Koselugo, AstraZeneca and Merck have a collaboration deal for Lynparza, which is ongoing.
AstraZeneca PLC Price and Consensus
AstraZeneca PLC price-consensus-chart | AstraZeneca PLC Quote
AZN's Zacks Rank & Stocks to Consider
AstraZeneca currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector include Editas Medicine (EDIT - Free Report) and ADMA Biologics (ADMA - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Editas Medicine’s loss per share have narrowed from $2.12 to $2.06 for 2025. During the same time, loss per share estimates for 2026 have widened from $1.02 to $1.05. Year to date, shares of EDIT have rallied 94.5%.
Editas Medicine’searnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 13.17%.
In the past 60 days, estimates for ADMA Biologics’ earnings per share have increased from 57 cents to 58 cents for 2025. During the same time, earnings per share estimates for 2026 have improved from 88 cents to 90 cents. Year to date, shares of ADMA have lost 4.6%.
ADMA Biologics’ earnings beat estimates in one of the trailing four quarters, matched once, and missed the same on the remaining two occasions, with the average negative surprise being 3.01%.