ASCIMINIB HYDROCHLORIDE - Generic Drug Details

What are the generic drug sources for asciminib hydrochloride and what is the scope of patent protection?

Asciminib hydrochloride is the generic ingredient in one branded drug marketed by Novartis and is included in one NDA. There are two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Asciminib hydrochloride has eighty-four patent family members in forty-nine countries.

One supplier is listed for this compound.

Summary for ASCIMINIB HYDROCHLORIDE

International Patents:	84
US Patents:	2
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	4
Clinical Trials:	16
Patent Applications:	2
DailyMed Link:	ASCIMINIB HYDROCHLORIDE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ASCIMINIB HYDROCHLORIDE

Constraining patent/regulatory exclusivity:

11,407,735

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for ASCIMINIB HYDROCHLORIDE

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Sarit Assouline	Phase 3
Novartis	Phase 3
Augusta University	Phase 2

See all ASCIMINIB HYDROCHLORIDE clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for ASCIMINIB HYDROCHLORIDE

L01EA	BCR-ABL tyrosine kinase inhibitors
L01E	PROTEIN KINASE INHIBITORS
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

US Patents and Regulatory Information for ASCIMINIB HYDROCHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Novartis	SCEMBLIX	asciminib hydrochloride	TABLET;ORAL	215358-001	Oct 29, 2021		RX	Yes	No	8,829,195			Y
Novartis	SCEMBLIX	asciminib hydrochloride	TABLET;ORAL	215358-003	Apr 18, 2024		RX	Yes	No	8,829,195			Y
Novartis	SCEMBLIX	asciminib hydrochloride	TABLET;ORAL	215358-001	Oct 29, 2021		RX	Yes	No

International Patents for ASCIMINIB HYDROCHLORIDE

Country	Patent Number	Title	Estimated Expiration
Cuba	24265	COMPUESTOS DERIVADOS DE BENZAMIDA PARA INHIBIR LA ACTIVIDAD DE ABL1, ABL2 Y BCR-ABL1, ÚTILES EN EL TRATAMIENTO DEL CÁNCER
Montenegro	03095	DERIVATI BENZAMIDA ZA INHIBIRANJE AKTIVNOSTI ABL1, ABL2 I BCR-ABL1 (BENZAMIDE DERIVATIVES FOR INHIBITING THE ACTIVITY OF ABL1, ABL2 AND BCR-ABL1)
Serbia	57177	DERIVATI BENZAMIDA ZA INHIBIRANJE AKTIVNOSTI ABL1, ABL2 I BCR-ABL1 (BENZAMIDE DERIVATIVES FOR INHIBITING THE ACTIVITY OF ABL1, ABL2 AND BCR-ABL1)

Supplementary Protection Certificates for ASCIMINIB HYDROCHLORIDE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2861579	301201	Netherlands		PRODUCT NAME: ASCIMINIB OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN, ZOALS ASCIMINIBHYDROCHLORIDE; REGISTRATION NO/DATE: EU/1/22/1670 20220826
2861579	2290039-3	Sweden		PRODUCT NAME: ASCIMINIB OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, SUCH AS ASCIMINIB HYDROCHLORIDE; REG. NO/DATE: EU/1/22/1670 20220826
2861579	2022C/548	Belgium		PRODUCT NAME: ASCIMINIB OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT ERVAN, ZOALS ASCIMINIB HYDROCHLORIDE; AUTHORISATION NUMBER AND DATE: EU/1/22/1670 20220826

ASCIMINIB HYDROCHLORIDE Market Analysis and Financial Projection

Market Dynamics and Financial Trajectory for Asciminib Hydrochloride (Scemblix)

Introduction

Asciminib hydrochloride, marketed as Scemblix, is a groundbreaking drug approved by the FDA for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). Here, we delve into the market dynamics and financial trajectory of this innovative medication.

Approval and Regulatory Framework

Asciminib received accelerated approval from the FDA in October 2021 for patients with Ph+ CML in chronic phase, previously treated with two or more tyrosine kinase inhibitors (TKIs), and for those with the T315I mutation[1][4].

The approval was facilitated through the Real-Time Oncology Review (RTOR) pilot program and various expedited programs, including priority review, breakthrough designation, fast track designation, and orphan drug designation. These designations highlight the drug's potential and the regulatory body's support for its rapid market entry[1].

Clinical Efficacy and Safety Profile

Asciminib has demonstrated superior efficacy compared to current standard-of-care TKIs. In the ASC4FIRST clinical trial, asciminib showed a higher major molecular response (MMR) rate of 68% at 48 weeks, compared to 49% for TKIs. Additionally, it exhibited a more favorable safety profile with fewer adverse events, drug interruptions, and discontinuations[3].

For patients with the T315I mutation, asciminib achieved an MMR rate of 42% by 24 weeks and 49% by 96 weeks, further solidifying its clinical value[1].

Market Positioning

Asciminib is poised to disrupt the CML treatment market due to its unique mechanism of action as an allosteric TKI, targeting the BCR::ABL1 kinase in a way that differs from traditional ATP-site inhibitors. This differentiation could lead to increased market share as it addresses resistance issues associated with current TKIs[2][3].

Competitive Landscape

The CML treatment market is dominated by TKIs such as imatinib and second-generation TKIs. However, asciminib's superior efficacy and safety profile position it as a potential first-line treatment option. The ASC4FIRST trial's results, showing asciminib's statistical superiority over current TKIs, are likely to influence prescribing practices and patient outcomes[3].

Patient and Physician Adoption

The favorable clinical data and safety profile of asciminib are expected to drive adoption among both patients and healthcare providers. As more patients achieve major molecular responses and deep molecular responses, the drug's reputation and usage are likely to increase. Physician preference, influenced by the lower rate of adverse events and treatment discontinuations, will also play a crucial role in its market penetration[3].

Financial Projections and Revenue Potential

Given its approval and positive clinical trial results, asciminib is expected to generate significant revenue for Novartis. The drug's orphan drug designation and other expedited programs indicate a potential for higher pricing, which could contribute to substantial revenue.

"Asciminib demonstrated superior efficacy compared to current standard-of-care targeted therapies, and was accompanied by a more favorable safety profile with fewer adverse events, drug interruptions, and drug discontinuations. This combination of potency and safety may enable more patients to achieve treatment-free remission, the ultimate goal of CML therapy," – Oreofe O. Odejide, MD, MPH, Dana-Farber Cancer Institute[3].

Cost and Access Considerations

The financial implications for patients and healthcare systems will be significant. Asciminib's pricing, while not explicitly stated, is likely to be competitive with other TKIs on the market. The drug's inclusion in programs like the 340B program, which helps manage financial responsibilities for pharmacies, could also impact its accessibility and affordability[5].

Long-Term Market Outlook

As researchers continue to follow participants to understand the long-term safety profile and outcomes of asciminib, the drug is likely to maintain its market position. Additional studies focusing on overall survival, progression-free survival, and treatment-free remission will further solidify its place in CML treatment protocols[3].

Challenges and Opportunities

While asciminib presents a significant opportunity in the CML market, it also faces challenges such as competition from established TKIs and the need for ongoing clinical trials to confirm long-term efficacy and safety. However, its unique mechanism of action and favorable clinical data position it well to overcome these challenges.

Key Takeaways

• Superior Efficacy: Asciminib has shown higher MMR rates and a more favorable safety profile compared to current TKIs.
• Market Positioning: Its unique allosteric mechanism positions it as a potential first-line treatment for Ph+ CML.
• Financial Projections: Expected to generate significant revenue for Novartis due to its orphan drug designation and favorable clinical data.
• Patient and Physician Adoption: Likely to see high adoption rates due to its superior clinical outcomes and safety profile.
• Long-Term Outlook: Ongoing studies will continue to support its market position and potential for treatment-free remission.

Frequently Asked Questions (FAQs)

Q1: What is asciminib hydrochloride used for? Asciminib hydrochloride, marketed as Scemblix, is used for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase, particularly in patients previously treated with two or more tyrosine kinase inhibitors (TKIs) or those with the T315I mutation.

Q2: How does asciminib differ from other TKIs? Asciminib is an allosteric TKI, targeting the BCR::ABL1 kinase in a way that differs from traditional ATP-site inhibitors. This unique mechanism of action helps in addressing resistance issues associated with current TKIs.

Q3: What are the common adverse reactions associated with asciminib? Common adverse reactions include upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. Laboratory abnormalities such as decreased platelet counts and increased triglycerides are also observed.

Q4: How is asciminib administered? Asciminib is administered orally, with recommended doses of 80 mg once daily or 40 mg twice daily for patients previously treated with two or more TKIs, and 200 mg twice daily for patients with the T315I mutation.

Q5: What are the long-term prospects for asciminib in the CML treatment market? Asciminib is expected to maintain a strong market position due to its superior efficacy and safety profile. Ongoing studies will continue to support its potential for treatment-free remission and long-term safety, further solidifying its place in CML treatment protocols.

Cited Sources:

1. FDA Approves Asciminib for Philadelphia Chromosome-Positive Chronic Myeloid Leukemia - FDA.
2. BCR::ABL1 Kinase N-LoBe Mutants Confer Moderate to High Degrees of Resistance to Tyrosine Kinase Inhibitors - ScienceDirect.
3. Asciminib May Be Safer, More Effective Treatment Option for People with Newly Diagnosed Chronic Myeloid Leukemia - ASCO.
4. Asciminib Hydrochloride (Scemblix) NDA Submission - FDA.
5. Asciminib Hydrochloride (Scemblix) - NCODA.