Voxelotor - Generic Drug Details

What are the generic sources for voxelotor and what is the scope of freedom to operate?

Voxelotor is the generic ingredient in one branded drug marketed by Global Blood Theraps and is included in two NDAs. There are eleven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Voxelotor has two hundred and seventy-seven patent family members in forty countries.

One supplier is listed for this compound.

Summary for voxelotor

International Patents:	277
US Patents:	11
Tradenames:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	48
Clinical Trials:	17
Patent Applications:	174
What excipients (inactive ingredients) are in voxelotor?	voxelotor excipients list
DailyMed Link:	voxelotor at DailyMed

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

Dosage:

TABLET;ORAL

Dosage:

TABLET, FOR SUSPENSION;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 voxelotor

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

Sponsor	Phase
Pfizer	Phase 1
Duke University	Phase 2
Global Blood Therapeutics (GBT)	Phase 2

See all voxelotor clinical trials

Pharmacology for voxelotor

Drug Class	Hemoglobin S Polymerization Inhibitor
Mechanism of Action	Cytochrome P450 3A4 Inhibitors Hemoglobin S Polymerization Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for voxelotor

B06AX	Other hematological agents
B06A	OTHER HEMATOLOGICAL AGENTS
B06	OTHER HEMATOLOGICAL AGENTS
B	Blood and blood forming organs

Paragraph IV (Patent) Challenges for VOXELOTOR

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
OXBRYTA	Tablets	voxelotor	300 mg and 500 mg	213137	2	2023-11-27
OXBRYTA	Tablets for Oral Suspension	voxelotor	300 mg	216157	1	2023-11-27

US Patents and Regulatory Information for voxelotor

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-002	Oct 14, 2022		DISCN	Yes	No
Global Blood Theraps	OXBRYTA	voxelotor	TABLET, FOR SUSPENSION;ORAL	216157-001	Dec 17, 2021		DISCN	Yes	No
Global Blood Theraps	OXBRYTA	voxelotor	TABLET;ORAL	213137-002	Oct 14, 2022		DISCN	Yes	No				Y
Global Blood Theraps	OXBRYTA	voxelotor	TABLET, FOR SUSPENSION;ORAL	216157-001	Dec 17, 2021		DISCN	Yes	No

EU/EMA Drug Approvals for voxelotor

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Pfizer Europe MA EEIG	Oxbryta	Voxelotor	EMEA/H/C/004869 Oxbryta is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.	Authorised	no	no	yes	2022-02-14

International Patents for voxelotor

Country	Patent Number	Title	Estimated Expiration
Lithuania	PA2022517
Japan	2021113225	２−ヒドロキシ−６−（（２−（１−イソプロピル−１Ｈ−ピラゾール−５−イル）ピリジン−３−イル）メトキシ）ベンズアルデヒドの遊離塩基の結晶多形 (CRYSTALLINE POLYMORPHS OF FREE BASE OF 2-HYDROXY-6-((2-(1-ISOPROPYL-1H-PYRAZOL-5-YL)PYRIDIN-3-YL)METHOXY)BENZALDEHYDE)
Taiwan	202037593	Compounds and uses thereof for the modulation of hemoglobin
South Korea	20160118204

Supplementary Protection Certificates for voxelotor

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2797416	122022000052	Germany		PRODUCT NAME: VOXELOTOR ODER EIN TAUTOMER ODER PHARMAZEUTISCH AKZEPTABLES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1622 20220214
2797416	LUC00276	Luxembourg		PRODUCT NAME: VOXELOTOR OR A TAUTOMER OR PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; AUTHORISATION NUMBER AND DATE: EU/1/21/1622 20220215
2797416	301191	Netherlands		PRODUCT NAME: VOXELOTOR OF EEN TAUTOMEER OF FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/21/1622 20220215
2797416	32/2022	Austria		PRODUCT NAME: VOXELOTOR ODER EIN TAUTOMER ODER PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1622 (MITTEILUNG) 20220215

Voxelotor Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Voxelotor (OXBRYTA)

Introduction

Voxelotor, marketed as OXBRYTA, is a significant drug in the treatment of sickle cell disease (SCD), a chronic and debilitating condition. However, recent developments have dramatically altered its market dynamics and financial trajectory.

Approval and Initial Success

OXBRYTA was approved by the FDA in November 2019 for the treatment of SCD in adults and children aged 12 years and older. Initially, it showed promising results, with Global Blood Therapeutics (GBT) reporting strong sales growth. In 2021, OXBRYTA generated $194.7 million in net sales, a 57% increase from the previous year, driven by expanding patient demand and label expansions[2].

Acquisition by Pfizer

In 2022, Pfizer acquired GBT for $5.4 billion, with OXBRYTA being a key driver of this acquisition. The expectation was that OXBRYTA would continue to grow and become a major revenue generator for Pfizer, with some analysts predicting sales could exceed $750 million by the end of the decade[3].

Voluntary Withdrawal

However, in September 2024, Pfizer announced the voluntary withdrawal of all lots of OXBRYTA from worldwide markets. This decision was based on new clinical data indicating an imbalance in vaso-occlusive crises and fatal events, suggesting that the overall benefit of OXBRYTA no longer outweighed the risk in the approved patient population[1][4].

Impact on Patients and Healthcare Providers

The withdrawal of OXBRYTA has left patients and healthcare providers scrambling for alternative treatment options. Sickle cell disease patients have historically been underserved, and the loss of this treatment adds to their challenges. Patients on OXBRYTA are advised to discuss alternative treatments with their physicians, highlighting the urgent need for other effective therapies[4].

Financial Implications

Despite the significant acquisition cost, the financial impact of OXBRYTA's withdrawal on Pfizer is expected to be modest. Pfizer recorded global sales of $328 million for OXBRYTA in 2023, which was below analyst expectations of around $400 million. However, the company does not anticipate a material impact on its fiscal year 2024 guidance[3][4].

Investor Sentiment

The withdrawal has raised concerns about Pfizer's business development track record, particularly given the substantial investment in acquiring GBT. Investors are questioning the company's ability to grow through the 2025-2030 period, especially with upcoming patent expirations and other challenges to current growth drivers[4].

Market Reaction

Pfizer's stock experienced a brief dip following the announcement but has since recovered. The modest financial impact and the company's diversified portfolio have helped mitigate the immediate effects on its stock price[3].

Regulatory and Competitive Landscape

The withdrawal of OXBRYTA may lead to increased scrutiny of other sickle cell disease treatments, including gene therapies recently approved by the FDA, such as bluebird bio’s Lyfgenia and Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy. This heightened scrutiny could affect the regulatory path for these newer therapies[4].

Alternative Treatments

The absence of OXBRYTA has opened opportunities for other biotech companies. For instance, Fulcrum Therapeutics, which is running a Phase Ib trial for pociredir, may see increased interest from patients seeking alternative treatments[3].

Clinical Trials and Expanded Access Programs

All active clinical trials and expanded access programs for OXBRYTA have been discontinued. Pfizer will continue to evaluate the new safety findings, which may provide insights into the drug's future or the development of similar therapies[1][4].

Conclusion on Market Dynamics

The market dynamics for voxelotor have shifted dramatically from a promising growth trajectory to a sudden and complete withdrawal. This change underscores the risks and uncertainties inherent in pharmaceutical development and the critical importance of ongoing safety monitoring.

Financial Trajectory

The financial trajectory of OXBRYTA, once expected to be a significant revenue generator, has been severely impacted. The drug's withdrawal will result in lost revenue and may affect Pfizer's long-term growth strategy, although the immediate financial impact is anticipated to be manageable.

Key Takeaways

• Voluntary Withdrawal: Pfizer has withdrawn OXBRYTA from global markets due to new safety data indicating an imbalance in vaso-occlusive crises and fatal events.
• Financial Impact: The withdrawal is expected to have a modest financial impact on Pfizer, despite the significant acquisition cost of GBT.
• Patient Impact: Patients and healthcare providers are left seeking alternative treatments, highlighting the need for other effective therapies for SCD.
• Regulatory Scrutiny: The withdrawal may increase regulatory scrutiny of other sickle cell disease treatments.
• Competitive Opportunities: Other biotech companies may benefit from the withdrawal as patients seek alternative treatments.

FAQs

What led to the withdrawal of OXBRYTA from the market?

The withdrawal was due to new clinical data showing an imbalance in vaso-occlusive crises and fatal events, indicating that the overall benefit of OXBRYTA no longer outweighed the risk in the approved patient population[1][4].

How did the acquisition of GBT by Pfizer impact OXBRYTA?

Pfizer acquired GBT for $5.4 billion in 2022, with OXBRYTA being a key driver of this acquisition. However, the subsequent withdrawal of OXBRYTA has raised questions about Pfizer's business development track record[3].

What are the alternative treatment options for SCD patients?

Patients can discuss alternative treatments with their physicians, including recently approved gene therapies such as bluebird bio’s Lyfgenia and Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy[4].

How has the market reacted to the withdrawal of OXBRYTA?

The market reaction has been modest, with Pfizer's stock experiencing a brief dip but recovering shortly after the announcement[3].

What is the expected impact on Pfizer's financial guidance?

The withdrawal of OXBRYTA is not expected to have a material impact on Pfizer's fiscal year 2024 guidance[1][4].

Sources

1. Pfizer Inc. - Pfizer Voluntarily Withdraws All Lots of Sickle Cell Disease Treatment OXBRYTA® (voxelotor) From Worldwide Markets[1].
2. Biospace - GBT Reports Fourth Quarter and Full Year 2021 Financial Results[2].
3. Biospace - Pfizer's Oxbryta Withdrawal Leaves Sickle Cell Community Scrambling, Investors Reeling[3].
4. Biospace - Pfizer Takes Sickle Cell Drug Oxbryta Off Global Market, Cites Risk of Death[4].