Berotralstat hydrochloride - Generic Drug Details

What are the generic drug sources for berotralstat hydrochloride and what is the scope of freedom to operate?

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

Berotralstat hydrochloride has eighty-six patent family members in thirty-seven countries.

One supplier is listed for this compound.

Summary for berotralstat hydrochloride

International Patents:	86
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	17
Clinical Trials:	4
Patent Applications:	5
What excipients (inactive ingredients) are in berotralstat hydrochloride?	berotralstat hydrochloride excipients list
DailyMed Link:	berotralstat hydrochloride at DailyMed

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

Dosage:

CAPSULE;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 berotralstat hydrochloride

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

Sponsor	Phase
Ionis Pharmaceuticals, Inc.	Phase 3
BioCryst Pharmaceuticals	Phase 3

See all berotralstat hydrochloride clinical trials

Pharmacology for berotralstat hydrochloride

Drug Class	Plasma Kallikrein Inhibitor
Mechanism of Action	Cytochrome P450 2D6 Inhibitors Cytochrome P450 3A4 Inhibitors Kallikrein Inhibitors P-Glycoprotein Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for berotralstat hydrochloride

B06AC	Drugs used in hereditary angioedema
B06A	OTHER HEMATOLOGICAL AGENTS
B06	OTHER HEMATOLOGICAL AGENTS
B	Blood and blood forming organs

Paragraph IV (Patent) Challenges for BEROTRALSTAT HYDROCHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
ORLADEYO	Capsules	berotralstat hydrochloride	110 mg and 150 mg	214094	1	2024-12-03

US Patents and Regulatory Information for berotralstat hydrochloride

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Biocryst	ORLADEYO	berotralstat hydrochloride	CAPSULE;ORAL	214094-001	Dec 3, 2020		RX	Yes	No
Biocryst	ORLADEYO	berotralstat hydrochloride	CAPSULE;ORAL	214094-002	Dec 3, 2020		RX	Yes	Yes
Biocryst	ORLADEYO	berotralstat hydrochloride	CAPSULE;ORAL	214094-001	Dec 3, 2020		RX	Yes	No
Biocryst	ORLADEYO	berotralstat hydrochloride	CAPSULE;ORAL	214094-002	Dec 3, 2020		RX	Yes	Yes
Biocryst	ORLADEYO	berotralstat hydrochloride	CAPSULE;ORAL	214094-001	Dec 3, 2020		RX	Yes	No

International Patents for berotralstat hydrochloride

Country	Patent Number	Title	Estimated Expiration
Eurasian Patent Organization	201691803	ИНГИБИТОРЫ КАЛЛИКРЕИНА ПЛАЗМЫ ЧЕЛОВЕКА
Israel	280785	מעכבים לקאליקראין של פלזמה אנושית (Human plasma kallikrein inhibitors)
European Patent Office	3113772	INHIBITEURS DE LA KALLICRÉINE PLASMATIQUE HUMAINE (HUMAN PLASMA KALLIKREIN INHIBITORS)
San Marino	T202100031
Brazil	112021008249

Supplementary Protection Certificates for berotralstat hydrochloride

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3113772	2021C/544	Belgium		PRODUCT NAME: BEROTRALSTAT ET SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; AUTHORISATION NUMBER AND DATE: EU/1/21/1544/001 ET 002 20210504
3113772	42/2021	Austria		PRODUCT NAME: BEROTRALSTAT UND PHARMAZEUTISCH ANNEHMBARE SALZE DAVON; REGISTRATION NO/DATE: EU/1/21/1544 20210504
3113772	C20210031 00416	Estonia		PRODUCT NAME: BEROTRALSTAAT;REG NO/DATE: EU/1/21/1544 04.05.2021
3113772	21C1048	France		PRODUCT NAME: BEROTRALSTAT ET SELS PHARMACEUTIQUEMENT ACCEPTABLES DE CELUI-CI; REGISTRATION NO/DATE: EU/1/21/1544 20210504
3113772	122021000062	Germany		PRODUCT NAME: BEROTRALSTAT ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/21/1544 20210430

Market Dynamics and Financial Trajectory for Berotralstat Hydrochloride (ORLADEYO)

Introduction to ORLADEYO

ORLADEYO, also known as berotralstat hydrochloride, is a groundbreaking oral medication developed by BioCryst Pharmaceuticals, Inc. for the prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older. Here, we delve into the market dynamics and financial trajectory of this significant pharmaceutical product.

Regulatory Approval and Market Authorization

ORLADEYO has received regulatory approvals in several key markets, including the United States and Canada. In Canada, Health Canada issued a Notice of Compliance for ORLADEYO on June 2, 2022, based on favorable quality, non-clinical, and clinical data[2].

Clinical Efficacy and Patient Outcomes

Berotralstat hydrochloride works by decreasing plasma kallikrein activity, thereby controlling excess bradykinin generation, which is a key factor in HAE attacks[4]. Real-world studies have shown high adherence and persistence rates among patients using ORLADEYO, similar to other long-term prophylactic (LTP) therapies. These studies also highlight statistically significant and sustained HAE attack rate reductions, regardless of the patient's C1-inhibitor (C1-INH) deficiency status[5].

Market Performance and Revenue Growth

2022 Financial Performance

In 2022, ORLADEYO demonstrated robust growth, with preliminary full-year net revenue of $251.6 million, representing a 105% year-over-year increase. The fourth quarter of 2022 saw net revenue of $70.7 million, a 53% year-over-year increase[3].

2023 Financial Performance

For the full year 2023, ORLADEYO's preliminary net revenue was $325 million, a 29% year-over-year increase. The fourth quarter of 2023 saw net revenue of $89.9 million, a 27% year-over-year increase. This growth was driven by strong patient adherence and the continued global expansion of the drug[1].

2024 Financial Outlook

BioCryst expects full-year 2024 global net ORLADEYO revenue to be between $380 million and $400 million. This projection is supported by the drug's consistent growth pattern, despite seasonal fluctuations in the first quarter due to prescription reauthorizations and the potential impact of the Inflation Reduction Act[1].

Peak Sales Projections

ORLADEYO is on a trajectory to achieve $1 billion in peak sales, a milestone that underscores its commercial success and the significant demand for effective HAE treatments. This projection is based on the drug's strong market performance and the expanding patient base switching to ORLADEYO for its excellent attack control and convenience[1][3].

Operating Expenses and Profitability

BioCryst has implemented a disciplined approach to capital allocation, which is expected to drive the company towards profitability. Operating expenses for 2024 are anticipated to remain flat, ranging between $365 million and $375 million. This disciplined approach, combined with the revenue growth from ORLADEYO, positions the company to achieve a full-year operating profit in 2024 and approach positive earnings per share (EPS) and positive cash flow in the second half of 2025[1].

Strategic Initiatives and Future Development

In addition to the success of ORLADEYO, BioCryst is actively developing other pipeline products, including BCX10013, an oral Factor D inhibitor. The company plans to out-license the late-stage development and commercialization of BCX10013 to a partner that can accelerate its development and maximize its commercial potential. This strategic move has led to a reduction in the size of the R&D organization and an accelerated timeline to profitability[1].

Patient Preference and Adherence

Real-world evidence has consistently shown that patients prefer oral LTP therapies like ORLADEYO due to their convenience and effectiveness. High adherence and persistence rates among ORLADEYO users further support its market position and contribute to its financial success[5].

Competitive Landscape

ORLADEYO's unique profile as an oral, once-daily prophylactic therapy sets it apart in the HAE treatment market. The drug's ability to reduce HAE attack rates significantly, regardless of C1-INH status, makes it a preferred choice for many patients and physicians. This differentiation is a key driver of its market dynamics and financial performance[5].

Challenges and Risks

Despite the strong market performance, BioCryst faces several challenges, including the ongoing COVID-19 pandemic, which could impact development, regulatory processes, and supply chains. Other risks include the company's ability to manage growth, compete effectively, and navigate international expansion. These factors could affect the company's financial results and forward-looking statements[1][3].

Key Takeaways

• Robust Revenue Growth: ORLADEYO has shown significant revenue growth, with a 29% year-over-year increase in 2023 and a projected range of $380-$400 million for 2024.
• Peak Sales Projections: The drug is on track to achieve $1 billion in peak sales.
• Operational Efficiency: BioCryst expects to achieve full-year operating profit in 2024 and approach positive EPS and cash flow in the second half of 2025.
• Patient Preference: High adherence and persistence rates, along with patient preference for oral LTP therapies, drive ORLADEYO's market success.
• Strategic Initiatives: The company is focusing on pipeline development and strategic partnerships to accelerate growth and profitability.

FAQs

Q: What is ORLADEYO used for?

ORLADEYO (berotralstat hydrochloride) is used for the prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older.

Q: How has ORLADEYO performed financially?

ORLADEYO has shown robust financial performance, with a preliminary full-year 2023 net revenue of $325 million, a 29% year-over-year increase, and a projected 2024 revenue range of $380-$400 million.

Q: What are the peak sales projections for ORLADEYO?

ORLADEYO is projected to achieve $1 billion in peak sales, driven by its strong market performance and expanding patient base.

Q: How does ORLADEYO compare to other HAE treatments?

ORLADEYO stands out as an oral, once-daily prophylactic therapy with high adherence and persistence rates, and it significantly reduces HAE attack rates regardless of C1-INH status.

Q: What are the key challenges facing BioCryst?

BioCryst faces challenges such as the ongoing COVID-19 pandemic, managing growth, competing effectively, and navigating international expansion, which could impact its financial results and forward-looking statements.

Sources

1. BioCryst Announces Preliminary Full Year 2023 ORLADEYO® (berotralstat) Net Revenue of $325 Million, Provides 2024 Guidance and Accelerated Path to Profitability. BioCryst Pharmaceuticals, Inc. January 5, 2024.
2. Summary Basis of Decision for Orladeyo. Health Canada. October 19, 2022.
3. BioCryst Announces Preliminary Full Year 2022 ORLADEYO® (berotralstat) Net Revenue and Provides Full Year 2023 ORLADEYO Net Revenue Guidance. BioCryst Pharmaceuticals, Inc. January 9, 2023.
4. Highlights of Prescribing Information - DailyMed. U.S. National Library of Medicine. December 4, 2020.
5. BioCryst Presents New Real-World Evidence Showing High Adherence and Persistence Rates with ORLADEYO (berotralstat). BioCryst Pharmaceuticals, Inc. October 25, 2024.