Metreleptin - Biologic Drug Details

Summary for metreleptin

Tradenames:	1
High Confidence Patents:	0
Applicants:	1
BLAs:	1
Suppliers: see list	2
Recent Clinical Trials:	See clinical trials for metreleptin

Recent Clinical Trials for metreleptin

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Amryt Pharma	Phase 1
University of Michigan	Phase 1
Amryt Pharma	Phase 3

See all metreleptin clinical trials

Pharmacology for metreleptin

Ingredient-type	Analogs/Derivatives Leptin
Established Pharmacologic Class	Leptin Analog

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. General brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for metreleptin Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for metreleptin Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for metreleptin Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for the Biologic Drug: Metreleptin

Introduction to Metreleptin

Metreleptin, marketed as MYALEPT® in the US and MYALEPTA® in the EU, is a recombinant analogue of human leptin. It is the first and only FDA-approved treatment for the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy[1].

Approval and Regulatory Milestones

• FDA Approval: MYALEPT was approved by the US FDA on February 25, 2014, as an adjunct to diet for the treatment of complications of leptin deficiency in patients with generalized lipodystrophy[1].
• European Approval: In addition to its US approval, metreleptin has been approved in Europe for generalized lipodystrophy and partial lipodystrophy[2].
• Health Canada Approval: Recently, MYALEPTA received approval from Health Canada for the treatment of patients with lipodystrophy, expanding its global reach[4].

Market Position and Competition

• Rare Disease Market: Metreleptin operates in the rare disease market, specifically targeting patients with lipodystrophy, a condition characterized by the loss or redistribution of body fat.
• Unique Selling Proposition: As the first and only approved therapy for leptin deficiency, MYALEPT holds a significant market position with little direct competition[1][2].

Financial Trajectory

Acquisition and Ownership

• AstraZeneca and Bristol-Myers Squibb: Initially, the Biologics License Application (BLA) for MYALEPT was transferred from Bristol-Myers Squibb to AstraZeneca as part of the acquisition of the diabetes alliance assets in February 2014[1].
• Amryt Pharma: In June 2019, Amryt Pharma acquired Aegerion Pharmaceuticals, which included the rights to metreleptin, significantly expanding Amryt’s portfolio in the rare disease market[2].
• Chiesi Group: In April 2023, Amryt Pharma was acquired by the Chiesi Group, further solidifying the global reach and commercial capabilities for MYALEPTA[4].

Revenue and Growth Opportunities

• Revenue Generation: The acquisition by Amryt Pharma brought in $136.5 million in built-in revenues from 2018, with multiple growth opportunities. Metreleptin, along with lomitapide (Lojuxta®/Juxtapid®), is expected to drive revenue growth and future profitability[2].
• Geographic Expansion: The approval and commercialization of MYALEPTA in Europe and Canada have expanded its market reach, contributing to revenue growth. The partnership with Medison has been instrumental in this expansion, particularly in Canada[4].

Operational Synergies and Cost Savings

• Synergies: The acquisition by Amryt Pharma is expected to deliver operational synergies of between $25 million and $40 million in 2020, rising further in subsequent years. These synergies are driven by the integration of Aegerion’s assets and the leveraging of Amryt’s commercial capabilities[2].

Pipeline and Future Development

• Potential Indications: Besides its current approvals, metreleptin is being explored as a potential treatment for partial lipodystrophy in the US, further expanding its market potential[2].
• Additional Products: The combined portfolio of Amryt Pharma, including metreleptin, lomitapide, and other development stage assets like AP101 for Epidermolysis Bullosa, positions the company for significant growth in the rare disease market[2].

Patient Support and Access

• Comprehensive Patient Support: Companies involved in the commercialization of metreleptin, such as AstraZeneca and Amryt Pharma, have committed to providing comprehensive patient support programs to ensure access to the treatment for eligible patients[1][5].
• Financial Assistance: Programs are in place to help commercially insured patients obtain 100% coverage of their monthly out-of-pocket costs for MYALEPT, up to an annual maximum[5].

Clinical Impact and Patient Outcomes

• Metabolic Disorder Treatment: Metreleptin directly impacts the underlying metabolic disorder of lipodystrophy, leading to effective symptom control. This treatment has been welcomed by clinicians and patients as it addresses a previously unmet medical need[4].
• Quality of Life: By treating the complications of leptin deficiency, metreleptin improves the physical, psychological, and emotional well-being of patients with lipodystrophy[4].

Challenges and Considerations

• Anti-Metreleptin Antibodies: There have been reports of anti-metreleptin antibodies with neutralizing activity in patients treated with MYALEPTA. The consequences of these antibodies are not well understood and require further monitoring[4].

Key Takeaways

• First and Only Approved Therapy: Metreleptin is the first FDA-approved treatment for leptin deficiency in patients with generalized lipodystrophy.
• Global Expansion: The drug has been approved in the US, Europe, and Canada, with ongoing efforts to expand its global reach.
• Revenue Growth: The acquisition by Amryt Pharma and subsequent acquisition by the Chiesi Group have positioned metreleptin for significant revenue growth.
• Operational Synergies: The integration of Aegerion’s assets with Amryt Pharma’s capabilities has led to meaningful operational synergies.
• Comprehensive Patient Support: Companies are committed to providing comprehensive patient support programs to ensure access to the treatment.

FAQs

What is metreleptin used for?

Metreleptin is used as a replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

Who are the current owners of the metreleptin franchise?

The metreleptin franchise is currently owned by the Chiesi Group, following the acquisition of Amryt Pharma in April 2023.

What are the potential future indications for metreleptin?

Besides its current approvals, metreleptin is being explored as a potential treatment for partial lipodystrophy in the US.

How does metreleptin impact patient outcomes?

Metreleptin directly impacts the underlying metabolic disorder of lipodystrophy, leading to effective symptom control and improving the physical, psychological, and emotional well-being of patients.

Are there any financial assistance programs available for patients taking metreleptin?

Yes, there are programs in place to help commercially insured patients obtain 100% coverage of their monthly out-of-pocket costs for MYALEPT, up to an annual maximum.

Sources

1. AstraZeneca Media Centre: "US FDA approves orphan drug MYALEPT™ (metreleptin for injection)".
2. MTS Partners: "Recommended Acquisition of Aegerion".
3. PTC Therapeutics, Inc.: "SEC Filing".
4. Biospace: "Chiesi Global Rare Diseases Announces Health Canada Approval of MYALEPTA".
5. Myalept.com: "SUPPORT | Myalept® (metreleptin) for injection".