Big pharma: Venetoclax Gets FDA's Breakthrough Therapy Designation In Higher Risk MDS

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https://markets.businessinsider.com...apy-designation-in-higher-risk-mds-1030628721

Jul. 21, 2021, 01:12 AM

AbbVie: Venetoclax Gets FDA's Breakthrough Therapy Designation In Higher Risk MDS​

 

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(RTTNews) - AbbVie (ABBV) announced Wednesday that the U.S. Food and Drug Administration granted a Breakthrough Therapy Designation or BTD to venetoclax (VENCLEXTA) in combination with azacitidine in Higher Risk Myelodysplastic Syndrome or MDS.

The BTD is granted for the potential treatment of adult patients with previously untreated intermediate-, high- and very high-risk MDS based on revised International Prognostic Scoring System or IPSS-R. This marks the sixth BTD granted to venetoclax.

FDA's BTD is intended to expedite the development and review of medications to treat a serious medical condition. It is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies.

MDS are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. venetoclax is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein.

 

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The company noted that the designation is supported by data from the Phase 1b M15-531 study. Venetoclax is also being investigated in combination with azacitidine for the treatment of MDS in the Phase Ib M15-522 study in patients with relapsed or refractory disease, and the Phase 3 randomized VERONA study in patients with newly diagnosed higher-risk MDS.

 

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VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

 

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https://www.businesswire.com/news/h...nt-of-Patients-With-Myelodysplastic-Syndromes

FDA Grants Breakthrough Therapy Designation for Venclexta in Combination With Azacitidine for the Treatment of Patients With Myelodysplastic Syndromes​

– Every year in the United States, approximately 10,000 people are diagnosed with myelodysplastic syndromes (MDS), and there remains a high unmet need for new treatment options –

– The designation is based on interim results from the Phase Ib M15-531 study investigating Venclexta plus azacitidine in people with previously untreated higher-risk MDS –

– This is the 11th Breakthrough Therapy Designation for Genentech’s hematology medicines and the sixth for Venclexta, demonstrating its potential across multiple blood cancers –

 

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– Every year in the United States, approximately 10,000 people are diagnosed with myelodysplastic syndromes (MDS), and there remains a high unmet need for new treatment options –

Angmoh the best: 10,000 in more than 300 million people diagnosed per year disease is considered valued high unmet need. In some other countries people with you die your business mentality will say no need to treat.

 

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A rare disease is generally considered to be a disease that affects fewer than 200,000 people in the United States at any given time. There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans.10 Jan 2020

Rare Diseases FAQ​

https://www.genome.gov › FAQ › Rare-Dise

 

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The incidence of MDS increases markedly with age, and the disease is most prevalent in individuals who are white and male. It is conservatively estimated that >10,000 new cases of MDS occur in the United States annually, and that ≥60,000 individuals with MDS currently reside in the country.
Risk Factors: Old age; Tobacco smoking

Epidemiology of Myelodysplastic Syndromes - NCBI - NIH​

 

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Myelodysplastic syndromes (MDS) are a rare group of blood disorders that occur as a result of disordered development of blood cells within the bone marrow.

Myelodysplastic Syndromes - NORD (National Organization ...​

 

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Is MDS a death sentence?

MDS is a potentially fatal disease; the common causes of death in a cohort of 216 MDS patients included bone marrow failure (infection/hemorrhage) and transformation to acute myeloid leukemia (AML). [4] Treatment of MDS can be challenging in these generally older patients.14 May 2011

When to Treat Myelodysplastic Syndromes - Cancer Network​

https://www.cancernetwork.com › view › when-treat-myel...

 

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Survival statistics for MDS

WPSS Risk Group	Median Survival	Risk of AML (within 5 years)
Low	5.5 years	14%
Intermediate	4 years	33%
High	2.2 years	54%
Very high	9 months	84%

1 more row
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22 Jan 2018

Survival Rates for Myelodysplastic Syndromes | MDS Prognosis​

 

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Survival statistics for MDS

WPSS Risk Group	Median Survival	Risk of AML (within 5 years)
Low	5.5 years	14%
Intermediate	4 years	33%
High	2.2 years	54%
Very high	9 months	84%

1 more row
•
22 Jan 2018

Survival Rates for Myelodysplastic Syndromes | MDS Prognosis​

Survival Statistics for Myelodysplastic Syndromes​

Survival statistics are a way for doctors and patients to get a general idea of the outlook (prognosis) for people with a certain type of cancer. They can’t tell you how long you will live, but they may help give you a better understanding about how likely it is that your treatment will be successful. Some people will want to know the survival statistics for their cancer, and some people won’t. If you don’t want to know, you don’t have to.

Median survival is one way to look at outcomes. It is the time after diagnosis at which half the patients in a certain group are still alive, and half have died. This is a middle value – half the patients live longer than this, and half do not live this long.

 

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Survival statistics don't tell the whole story​

Survival stats are often based on previous outcomes of large numbers of people who had the disease, but they can’t predict what will happen in any particular person’s case. There are some limitations to remember:

• The numbers below are based on patients diagnosed with a myelodysplastic syndrome (MDS) some time ago. Improvements in treatment since these numbers were gathered may result in a better outlook for people now being diagnosed with MDS.
• These numbers are based on prognostic scores that take into account certain factors, such as the type of MDS, the results of certain blood tests, and whether the abnormal cells have certain chromosome changes. But other factors might also affect a person’s outlook, such as the patient’s age and health, and how well the disease responds to treatment.

 

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Survival statistics for MDS

WPSS Risk Group	Median Survival	Risk of AML (within 5 years)
Low	5.5 years	14%
Intermediate	4 years	33%
High	2.2 years	54%
Very high	9 months	84%

1 more row
•
22 Jan 2018

Survival Rates for Myelodysplastic Syndromes | MDS Prognosis​

The WHO Prognostic Scoring System (WPSS) risk groups can also be used to predict outcome – both median survival and the chance that the MDS will transform into acute myeloid leukemia (AML) within 5 years. These statistics were published in 2007 based on patients diagnosed between 1982 and 2004.