Alkermes (ALKS) wins orphan drug designations in U.S. and Europe for alixorexton

Rhea-AI Filing Summary

Alkermes plc reported that alixorexton, its investigational orexin 2 receptor agonist for sleep disorders, has received orphan drug designations in both the U.S. and Europe. The FDA granted orphan status for idiopathic hypersomnia, while the European Commission granted orphan status for narcolepsy.

Alixorexton is in phase 3 Brilliance studies for narcolepsy types 1 and 2 and in the phase 2 Vibrance-3 study for idiopathic hypersomnia. Orphan designation can provide tax credits, reduced regulatory fees and market exclusivity, supporting development of this potential treatment for rare, chronic neurological conditions.

Insights

Biopharmaceutical sector analyst

Orphan designations strengthen alixorexton’s regulatory and commercial positioning.

The FDA and European Commission have granted orphan drug designations to alixorexton for idiopathic hypersomnia and narcolepsy. This status can provide tax credits, reduced fees and multi‑year market exclusivity if the drug is ultimately approved.

Alixorexton is already in late-stage development, with phase 3 Brilliance studies in narcolepsy types 1 and 2 and a phase 2 Vibrance-3 trial in idiopathic hypersomnia. These designations align with its focus on rare sleep disorders and may enhance the economics of successful approval.

The company highlights prior Breakthrough Therapy designation for narcolepsy type 1, adding another layer of regulatory support. Actual impact will depend on future clinical results, regulatory reviews and completion of ongoing studies referenced by their clinical trial identifiers.

8-K Event Classification

3 items: 7.01, 8.01, 9.01

3 items

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Item 8.01 Other Events Other

Voluntary disclosure of events the company deems important to shareholders but not covered by other items.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, and exhibit attachments filed with this report.

Key Figures

U.S. market exclusivity: 7 years EU market exclusivity: up to 10 years IH prevalence in U.S.: 40,000 people +2 more

5 metrics

U.S. market exclusivity 7 years Potential orphan drug market exclusivity in United States, if approved

EU market exclusivity up to 10 years Potential orphan drug market exclusivity in European Union, if approved

IH prevalence in U.S. 40,000 people Estimated number of idiopathic hypersomnia patients in the United States

Clinical phase – narcolepsy Phase 3 Brilliance Studies in narcolepsy type 1 and type 2

Clinical phase – idiopathic hypersomnia Phase 2 Vibrance-3 study in adults with idiopathic hypersomnia

Key Terms

orphan drug designation, Breakthrough Therapy designation, orexin 2 receptor (OX2R) agonist, idiopathic hypersomnia, +2 more

6 terms

orphan drug designation regulatory

"was recently granted orphan drug designations (ODD) from leading regulatory bodies in the U.S. and Europe"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

Breakthrough Therapy designation regulatory

"Alixorexton previously received Breakthrough Therapy designation for the treatment of NT1 from the FDA"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

orexin 2 receptor (OX2R) agonist medical

"a novel, investigational, oral, selective orexin 2 receptor (OX2R) agonist in development"

idiopathic hypersomnia medical

"Idiopathic hypersomnia (IH) is a rare, chronic, neurological sleep disorder"

A chronic sleep disorder that causes extreme daytime sleepiness and prolonged, unrefreshing sleep despite adequate night sleep, with no identifiable medical or neurological cause. Think of it like a phone battery that won’t hold charge: sufferers remain tired and less productive even after long rest. For investors, idiopathic hypersomnia matters because it creates demand for treatments, diagnostics and supportive services, influencing market opportunities and regulatory pathways in sleep medicine.

narcolepsy type 1 (NT1) medical

"in development for the treatment of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH)"

Brilliance Studies medical

"Alixorexton is currently being evaluated in the phase 3 Brilliance Studies in adults with NT1 and NT2"

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Understanding 8-K Material Events →

0001520262FalseAlkermes plc.00015202622026-06-152026-06-15

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF THE

SECURITIES EXCHANGE ACT OF 1934

Date of Report (Date of earliest event reported): June 15, 2026

ALKERMES PUBLIC LIMITED COMPANY

(Exact name of registrant as specified in its charter)

Ireland		001-35299				98-1007018
(State or other jurisdiction		(Commission				(IRS Employer
of incorporation)		File Number)				Identification No.)
Connaught House, 1 Burlington Road
Dublin 4, Ireland D04 C5Y6
(Address of principal executive offices)

Registrant's telephone number, including area code: + 353-1-772-8000

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

☐		Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐		Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐		Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐		Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class		Trading Symbol(s)		Name of each exchange on which registered
Ordinary shares, $0.01 par value		ALKS		Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 7.01 Regulation FD Disclosure.

On June 15, 2026, Alkermes plc (the “Company”) issued a press release announcing the grant of orphan drug designations to alixorexton, as described in Item 8.01 of this Current Report on Form 8-K. A copy of the related press release is furnished herewith as Exhibit 99.1 and is incorporated herein by reference.

The information in this Item 7.01, and in Exhibit 99.1 furnished herewith, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, or incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.

Item 8.01 Other Events.

On June 15, 2026, the Company announced that the U.S. Food and Drug Administration has granted orphan drug designation to alixorexton for the treatment of idiopathic hypersomnia, and the European Commission has granted orphan drug designation to alixorexton for the treatment of narcolepsy.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

EXHIBIT INDEX

Exhibit No.		Description
99.1		Press release issued by Alkermes plc dated June 15, 2026.
104		Cover page interactive data file (embedded within the Inline XBRL document).

2

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	ALKERMES PLC
Date: June 15, 2026	By:		/s/ David J. Gaffin
			David J. Gaffin
			Secretary

3

Exhibit 99.1

	Alkermes Contacts:
	For Investors:	Sandy Coombs, +1 781 609 6377
	For Media:	Gretchen Murphy,+1 781 609 6419

Alkermes Announces Orphan Drug Designations for Alixorexton in the U.S. and Europe

DUBLIN, June 15, 2026 -- Alkermes plc (Nasdaq: ALKS) today announced that alixorexton, a novel, investigational, oral, selective orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH), was recently granted orphan drug designations (ODD) from leading regulatory bodies in the U.S. and Europe. The U.S. Food and Drug Administration (FDA) has granted ODD to alixorexton for the treatment of IH, and the European Commission has granted ODD to alixorexton for the treatment of narcolepsy.

“Narcolepsy and idiopathic hypersomnia are rare, chronic neurological conditions for which significant unmet need remains. These orphan drug designations represent important milestones for the alixorexton program and underscore its potential, if approved, to advance care for the narcolepsy and idiopathic hypersomnia patient communities,” said Craig Hopkinson, M.D., (MBChB), Chief Medical Officer and Executive Vice President, Research & Development at Alkermes. “Alixorexton’s phase 2 clinical trial results in narcolepsy type 1 and type 2 underscore its potential to become a differentiated treatment option. We look forward to continuing our momentum in the alixorexton development program as we enroll the phase 3 Brilliance Studies and work to complete the Vibrance-3 phase 2 study in IH this year.”

Orphan drug designation supports the development of medicines intended to treat rare diseases. In the United States, orphan drug designation qualifies the drug developer for a variety of development incentives, including tax credits for qualified clinical testing, exemptions from certain FDA application fees, and seven years of market exclusivity, if approved. In the European Union, orphan designation provides incentives that may include protocol assistance, reduced regulatory fees, and up to 10 years of market exclusivity, if approved.

Alixorexton previously received Breakthrough Therapy designation for the treatment of NT1 from the FDA. Alixorexton is currently being evaluated in the phase 3 Brilliance Studies in adults with NT1 and NT2, and in the phase 2 Vibrance-3 study in adults with IH (Brilliance NT1 – Study 302: NCT07455383; Brilliance NT2 – Study 303: NCT07502443; Brilliance NT1 – Study 304: NCT07540897; Vibrance-3: NCT06843590).

About Narcolepsy

Narcolepsy is a rare, chronic, neurological disorder that affects the brain’s ability to regulate the sleep/wake cycle. Excessive daytime sleepiness is the hallmark symptom of narcolepsy; additional symptoms can include sleep paralysis, sleep-related hallucinations and disturbed nighttime sleep.1 There are two types of narcolepsy: narcolepsy type 1 is characterized by the loss of orexin-producing neurons, and is also associated with cataplexy, a sudden loss of muscle control while a person is awake, often triggered by strong emotions.2 The underlying neuropathology of narcolepsy type 2 remains to be fully elucidated; however the orexin pathway may play an important role.3

About Idiopathic Hypersomnia

Idiopathic hypersomnia (IH) is a rare, chronic, neurological sleep disorder characterized by excessive daytime sleepiness despite normal sleep durations.4,5 Additional common symptoms can include severe sleep inertia (individuals may feel groggy or disoriented for prolonged periods after waking up), unrefreshing naps, fatigue and cognitive dysfunction.6,7 The underlying neuropathology of idiopathic hypersomnia is unknown.4 IH affects an estimated 40,000 people in the U.S.8

About Alixorexton

Alixorexton (formerly referred to as ALKS 2680) is a novel, investigational, oral, selective orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH). Orexin, a neuropeptide produced in the lateral hypothalamus, is considered to be the master regulator of wakefulness due to its activation of multiple, downstream wake-promoting pathways that project widely throughout the brain.9 Targeting the orexin system may address excessive daytime sleepiness across hypersomnolence disorders, whether or not deficient orexin signaling is the underlying cause of disease.10  Alixorexton is currently being evaluated in the phase 3 Brilliance Studies in patients with NT1 and NT2, and in the phase 2 Vibrance-3 study in patients with IH. The U.S. Food and Drug Administration (FDA) has granted alixorexton Breakthrough Therapy designation for the treatment of NT1 and Orphan Drug Designation (ODD) for the treatment of IH. The European Commission has granted ODD to alixorexton for the treatment of narcolepsy.

About Alkermes plc

Alkermes plc (Nasdaq: ALKS), a mid-cap growth and value equity, is a global biopharmaceutical company that seeks to develop innovative medicines in the field of neuroscience. The company has a portfolio of proprietary commercial products for the treatment of alcohol dependence, opioid dependence, schizophrenia, bipolar I disorder and narcolepsy. Alkermes’ pipeline includes late-stage clinical candidates in development for narcolepsy and idiopathic hypersomnia, and orexin 2 receptor agonists in early clinical development for other neurological disorders, including attention-deficit hyperactivity disorder (ADHD) and fatigue associated with multiple sclerosis and Parkinson’s disease. Headquartered in Ireland, Alkermes also has a corporate office and research and development center in Massachusetts and a manufacturing facility in Ohio. For more information, please visit Alkermes’ website at www.alkermes.com.

Note Regarding Forward-Looking Statements

Certain statements set forth in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the potential therapeutic and commercial value of alixorexton, its potential regulatory approval, and timelines for its continued clinical development. The company cautions that forward-looking statements are inherently uncertain. Although the company believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, the forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those expressed or implied

in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: initial clinical results for alixorexton may not be predictive of results of future stages of ongoing clinical studies, future clinical studies or real-world results; ongoing or future clinical studies for alixorexton may not be initiated or completed on expected timelines or at all; alixorexton may be shown to be ineffective or unsafe; the FDA may not agree with the company’s regulatory strategies or components of its development program for alixorexton, including clinical trial designs, conduct and methodologies; potential changes in the cost, scope and duration of the alixorexton development program; and those risks and uncertainties described under the heading “Risk Factors” in the company’s Annual Report on Form 10-K for the year ended Dec. 31, 2025 and in subsequent filings made by the company with the U.S. Securities and Exchange Commission (SEC), which are available on the SEC’s website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by law, the company disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release.

‌1 Ruoff C, Rye D. The ICSD-3 and DSM-5 guidelines for diagnosing narcolepsy: clinical relevance and practicality. Curr Med Res Opin. 2016;32(10):1611-1622. doi:10.1080/03007995.2016.1208643

2 Dauvilliers Y, Siegel JM, Lopez R, Torontali ZA, Peever JH. Cataplexy--clinical aspects, pathophysiology and management strategy. Nat Rev Neurol. 2014;10(7):386-395. doi:10.1038/nrneurol.2014.97

3 Bassetti CLA, Adamantidis A, Burdakov D, et al. Narcolepsy - clinical spectrum, aetiopathophysiology, diagnosis and treatment. Nat Rev Neurol. 2019;15(9):519-539. doi:10.1038/s41582-019-0226-9

4 Trotti LM, Arnulf I. Idiopathic Hypersomnia and Other Hypersomnia Syndromes. Neurotherapeutics. 2021;18(1):20-31. doi:10.1007/s13311-020-00919-1

5 American Academy of Sleep Medicine. The International Classification of Sleep Disorders. Third Edition (ICSD-3). 2014.

6 Trotti LM. Waking up is the hardest thing I do all day: Sleep inertia and sleep drunkenness. Sleep Med Rev 2016.

7 Vernet C, Leu-Semenescu S, Buzare MA, Arnulf I. Subjective symptoms in idiopathic hypersomnia: beyond excessive sleepiness. J Sleep Res. 2010;19:525–534.

8 Acquavella et al. Prevalence of narcolepsy and other sleep disorders and frequency of diagnostic tests from 2013-2016 in insured patients actively seeking care. J Clin Sleep Med. 16:1255 (2020).

9 Buysse, D. Diagnosis and assessment of sleep and circadian rhythm disorders. Journal of Psychiatric Practice. 2005; 11(2):102-115

10 Ten-Blanco M, Flores A, Cristino L, Pereda-Perez I. Targeting the orexin/hypocretin system for the treatment of neuropsychiatric and neurodegenerative diseases: From animal to clinical studies. Frontiers in Neuroendocrinology. 2023;69(101066). https://www.sciencedirect.com/science/article/pii/S0091302223000146

FAQ

What did Alkermes (ALKS) announce about alixorexton in this 8-K?

Alkermes announced that alixorexton received orphan drug designations in the U.S. and Europe. The FDA granted orphan status for idiopathic hypersomnia, and the European Commission granted orphan status for narcolepsy, supporting development for these rare sleep disorders.

Which conditions are covered by alixorexton’s orphan drug designations?

Alixorexton’s orphan designations cover idiopathic hypersomnia in the U.S. and narcolepsy in Europe. The drug is also being developed for narcolepsy type 1 and type 2, reflecting a broader focus on rare hypersomnolence disorders with significant unmet need.

What stage of clinical development is Alkermes’ alixorexton in?

Alixorexton is in phase 3 Brilliance Studies for narcolepsy types 1 and 2 and a phase 2 Vibrance-3 study for idiopathic hypersomnia. These late- and mid-stage trials will help determine its safety and effectiveness across these rare sleep disorders.

What benefits can orphan drug designation provide to Alkermes’ alixorexton?

Orphan drug designation can provide tax credits, reduced regulatory fees and market exclusivity periods if the drug is approved. In the U.S., potential exclusivity can last seven years, while in the European Union it can extend up to ten years after approval.

Does alixorexton have any other special regulatory status besides orphan designation?

Yes. Alixorexton previously received Breakthrough Therapy designation from the FDA for narcolepsy type 1. This status is intended to expedite development and review for serious conditions when early clinical data suggest substantial improvement over existing therapies.

What type of drug is alixorexton and how does it work?

Alixorexton is a novel, oral, selective orexin 2 receptor (OX2R) agonist. It targets the orexin system, a key regulator of wakefulness, aiming to address excessive daytime sleepiness across narcolepsy and idiopathic hypersomnia by activating wake-promoting pathways in the brain.

What therapeutic areas does Alkermes (ALKS) focus on beyond alixorexton?

Alkermes focuses on neuroscience, with commercial products for alcohol and opioid dependence, schizophrenia, bipolar I disorder and narcolepsy. Its pipeline includes late-stage candidates for narcolepsy and idiopathic hypersomnia and early-stage orexin 2 receptor agonists for ADHD and fatigue in neurological diseases.

Filing Exhibits & Attachments

2 documents

Press Releases

• EX-99.1 EX-99.1 32.9 KB

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA WITH EMBEDDED LINKBASES DOCUMENT 30.1 KB