Sacrosidase - Biologic Drug Details

Summary for sacrosidase

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

Recent Clinical Trials for sacrosidase

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
QOL Medical, LLC	Phase 2
QOL Medical, LLC	Phase 4

See all sacrosidase clinical trials

Pharmacology for sacrosidase

Established Pharmacologic Class

Sucrose-specific Enzyme

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 sacrosidase Derived from Brand-Side Litigation

No patents found based on brand-side litigation

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

No patents found based on company disclosures

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

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for the Biologic Drug: Sacrosidase

Introduction

Sacrosidase, marketed as Sucraid, is a biologic drug used to treat Congenital Sucrase-Isomaltase Deficiency (CSID), a rare gastrointestinal genetic disorder. Understanding the market dynamics and financial trajectory of this drug is crucial for stakeholders, including patients, healthcare providers, and pharmaceutical companies.

What is Sacrosidase?

Sacrosidase is an enzyme replacement therapy that helps patients with CSID digest sucrose, a common sugar found in many foods. It is manufactured by QOL Medical, LLC, a specialty biopharmaceutical company focused on rare diseases and gastrointestinal disorders[4].

Market Context: Biologics and Rare Diseases

Biologics, including sacrosidase, are a growing segment of the pharmaceutical market. They now represent 42% of the total medicines market, up from 30% in 2014. This growth is driven by their effectiveness in treating complex and rare diseases[3].

Regulatory Environment

The FDA plays a critical role in the approval and monitoring of biologic drugs like sacrosidase. In 2022, the FDA approved Sucraid in single-use containers for patients over 33 pounds (15 kg) with CSID, enhancing patient convenience and adherence to treatment[4].

Supply and Shortage Issues

Historically, sacrosidase has faced supply shortages, which have significant implications for patients and healthcare providers. The FDA has worked closely with QOL Medical to mitigate these shortages, prioritizing access for patients with severe CSID who cannot be managed with diet alone[1].

Financial Assistance Programs

Given the financial burden of managing a rare disease, QOL Medical offers several financial assistance programs through SucraidASSIST™. These programs include copay and deductible savings, patient assistance for those below 200% of the Federal Poverty Level, and other forms of support to make the medication more affordable for eligible patients[5].

Market Access and Distribution

Sacrosidase is distributed through various channels, including specialty pharmacies and participating eligible pharmacies in the United States, Puerto Rico, and U.S. territories. The medication is typically prescribed by a physician and must be dispensed through approved channels to ensure patient safety and compliance[5].

Pricing and Reimbursement

The pricing of sacrosidase can be a significant factor in its financial trajectory. Patients with commercial insurance may benefit from the Sucraid Savings Program, which reduces the patient's responsibility to $5 per prescription, with a maximum annual benefit of up to $10,000. However, this program is not available for patients covered by federal, state, or other government health insurance programs[5].

Impact of REMS Programs

Risk Evaluation and Mitigation Strategies (REMS) programs, while not directly applicable to sacrosidase, can influence the broader biologic market. REMS programs can add complexity and burden to the prescribing process, potentially affecting patient access to certain therapies. However, they also ensure that patients are adequately informed and protected from potential risks associated with biologic drugs[2].

Patient and Healthcare Provider Perspectives

For patients with CSID, sacrosidase is a critical treatment that significantly improves their quality of life. The new single-use containers approved by the FDA have made it easier for patients to manage their medication. Healthcare providers also benefit from these advancements, as they can now offer more convenient and effective treatment options for their patients[4].

Financial Trajectory

The financial trajectory of sacrosidase is influenced by several factors, including market demand, competition from other treatments, and reimbursement policies. Given the niche market for CSID, the demand for sacrosidase is relatively stable, and the drug's approval in single-use containers has likely increased its market share. However, the overall financial performance is also dependent on the company's ability to manage supply chains and mitigate shortages[1][4].

Competitive Landscape

While sacrosidase is a unique treatment for CSID, the broader biologic market is becoming increasingly competitive with the emergence of biosimilars. By the end of 2019, 17% of the biologics market was accessible to biosimilars, and this trend is expected to continue. However, sacrosidase's niche market position and the specific nature of CSID make it less susceptible to immediate biosimilar competition[3].

Future Outlook

The future outlook for sacrosidase is positive, driven by ongoing improvements in patient care and the commitment of QOL Medical to enhance treatment options. The FDA's continued support and the expansion of financial assistance programs will likely contribute to increased patient access and adherence to treatment.

"FDA’s approval of Sucraid in single-use containers will make it easier for patients to manage their medication," said Weng Tao, MD, PhD, COO of QOL Medical, LLC. "We are always looking for ways to improve their quality of life."[4]

Key Takeaways

• Market Niche: Sacrosidase is a biologic drug treating a rare gastrointestinal genetic disorder, Congenital Sucrase-Isomaltase Deficiency (CSID).
• Regulatory Approval: FDA-approved in single-use containers to enhance patient convenience.
• Financial Assistance: Various programs available to make the medication more affordable.
• Supply Chain: Historical shortages have been mitigated through FDA and manufacturer collaboration.
• Pricing and Reimbursement: Commercial insurance programs and patient assistance available.
• Competitive Landscape: Less susceptible to biosimilar competition due to its niche market.

FAQs

Q: What is sacrosidase used for?

A: Sacrosidase, marketed as Sucraid, is used to treat the symptoms associated with sucrose ingestion in patients with Congenital Sucrase-Isomaltase Deficiency (CSID).

Q: Why has sacrosidase faced supply shortages?

A: Sacrosidase has faced supply shortages due to manufacturing issues, which the FDA and QOL Medical have worked to mitigate by prioritizing access for severe cases.

Q: What financial assistance programs are available for sacrosidase?

A: QOL Medical offers the SucraidASSIST™ program, which includes copay and deductible savings, patient assistance for those below 200% of the Federal Poverty Level, and other forms of support.

Q: How does the FDA approval of single-use containers impact patients?

A: The FDA approval of single-use containers makes it easier for patients to manage their medication, improving their quality of life.

Q: Is sacrosidase susceptible to biosimilar competition?

A: Given its niche market position and the specific nature of CSID, sacrosidase is less susceptible to immediate biosimilar competition.

Sources:

1. FDA, "Related Information for Sacrosidase Oral Solution - FDA," May 2, 2018.
2. Pharmaceutical Commerce, "With a Few Stumbles, REMS Begins to Hit Its Stride," April 22, 2011.
3. FTC, "Biologics Market Dynamics: Setting the Stage for Biosimilars," March 9, 2020.
4. Biospace, "QOL Medical, LLC receives FDA approval of Sucraid® (sacrosidase) Oral Solution single-use containers for patients with Congenital Sucrase-Isomaltase Deficiency (CSID)," September 6, 2022.
5. Sucraid, "SucraidASSIST™- Financial Support Services for Patients," Accessed December 19, 2024.