SteroTherapeutics Receives FDA Orphan-Drug Designation

PHILADELPHIA, April 04, 2018 — SteroTherapeutics, a privately held biopharmaceutical company developing therapies focused on metabolic diseases including non-alcoholic steatohepatitis (NASH), announced today that the U.S. Food and Drug Administration has granted orphan drug designation for ST-002 in the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis and hyperglycemia in patients with Cushing’s syndrome.

“We are pursuing a drug that has a very real potential to become the optimal agent of choice and a standard of care for these Cushing’s patients,” said Manohar Katakam Ph. D., CEO of SteroTherapeutics. “Our clinical trial will target multiple critical metabolic-related outcomes including the reduction of triglycerides, insulin resistance, weight loss, and the prevention and/or abrogation of hepatic steatosis and fibrosis.”

“The FDA’s orphan-drug designation for Fluasterone highlights the significant unmet and underserved needs for treatment in these individuals,” added Dr. Katakam. “We look forward to realizing the benefits and promise of this potential for Fluasterone in Cushing’s syndrome patients.”

The Orphan Drug Act became law in 1983. Fewer than 5,000 applicants have received this designation, according to the FDA website. Rare conditions are often described as orphan diseases or disorders when there are few or no treatment options. There are approximately 7,000 known orphan diseases.

The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

The designation allows the sponsor of the drug to be eligible for various incentives, including a seven-year period of U.S. marketing exclusivity upon regulatory approval of the drug, as well as tax credits for clinical research costs, annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

Cushing syndrome occurs when a patient’s body is exposed to high levels of the hormone cortisol over a long period of time (chronic hypercortisolemia) . Cushing syndrome, sometimes called hypercortisolism, affects 15,000 to 20,000 patients in the United States.

Too much cortisol can produce some of the hallmark signs of Cushing syndrome — a fatty hump between a patient’s shoulders, a rounded face, and pink or purple stretch marks on the skin. Cushing syndrome can also result in high blood pressure, bone loss and upper body obesity, increased fat around the neck, and relatively slender arms and legs. Diabetes is frequently a complication found in Cushing’s syndrome patients. These patients also develop nonalcoholic fatty disease and steatosis as a result of the chronic hypercortisolism.

About SteroTherapeutics

SteroTherapeutics, a Philadelphia, PA area based company, is focused on developing novel therapies for significant unmet needs in metabolic disease including liver diseases.

SteroTherapeutics lead products have been proven in previous human studies to possess a strong safety profile and established mechanisms of action. The company’s strategic intent is to focus on understanding disease pathways and how to safely treat and restore an optimal quality of life.  SteroTherapeutics is managed by a veteran team that has significant experience in the pharmaceutical and biotechnology industry. The team has specific experiences in the development, manufacturing and commercialization of small molecule and biologics based products.

INVESTOR RELATIONS CONTACT:
Tony Schor, Investor Awareness, Inc. on behalf of
SteroTherapeutics, LLC
tschor@sterotx.com/ (847) 945-2222 ext. 221

From https://www.econotimes.com/SteroTherapeutics-Receives-FDA-Orphan-Drug-Designation-1236099

Bruising easily and gaining weight? Don’t mistake high blood pressure for this syndrome

By Olivia Lerche June 30th, 2017

Cushing’s sydnrome [sic]: Condition can have the same symptoms as high blood pressure

The condition is a hormonal disorder caused by prolonged exposure to the hormone cortisol – which can be caused by taking steroids. Cortisol regulates metabolism and immune response in the body.

Other people develop Cushing’s syndrome because their bodies produce too much cortisol.

It is most common in adults aged between 20 and 50 although women are almost three time [sic] as likely to be diagnosed.

While the condition is rare and only affects around one in every 50,000 people – the syndrome can affect people with type 2 diabetes, obese and have poorly controlled blood sugar levels.

The condition is also more common in people with high blood pressure.

Cushing’s syndrome – also called hypercortisolism – can cause a number of symptoms.These can include:
Weight gain
Thinning skin which can bruise easily
Reddish-purple stretch marks on the arms, legs, breasts, thighs, stomach and buttocks
It can also cause the face to become rounder – causing fat to deposit on the face
Muscle or bone weakness is also a sign of the condition
A loss of libido – decreased interest in sex – is also a symptom

Cushing's sydnrome: Condition can have the same symptoms as high blood pressureGETTY

Cushing’s sydnrome [sic]: Condition has similar symptoms as high blood pressure and metabolic syndrome

However, other symptoms can include excess hair on the face, irregular periods, severe fatigue, high blood pressure, high blood sugar irritability or depression and even a fatty deposit between the shoulders.The National Institute of Diabetes and Digestive and Kidney Diseases said: “Metabolic syndrome – a combination of problems that includes excess weight around the waist, high blood pressure, abnormal levels of cholesterol and triglycerides in the blood, and insulin resistance-also mimics the symptoms of Cushing’s syndrome.”Cushing’s syndrome often develops as a side effect of treatment with corticosteroids.

Corticosteroids are widely used to reduce inflammation and treat autoimmune conditions  – where the immune system malfunctions and attacks healthy tissue – such as rheumatoid arthritis, Crohn’s disease and lupus.

Cushing's sydnrome: Condition can have the same symptoms as high blood pressureGETTY

Cushing’s sydnrome [sic]: Condition can have the same symptoms as high blood pressure

Metabolic syndrome also mimics the symptoms of Cushing’s syndrome

However, the condition can be hard to diagnose because of the similarity to high blood pressure.To diagnose the disease, patients will usually need to have a saliva test, urine test and blood test to measure cortisol levels in the body.To treat the condition, patients will usually have to decrease the levels of steroids they are taking.

However, there are complications if the condition is left untreated.

It can lead to high blood pressure and increase the risk of heart disease and stroke.

Other Diseases

forums

Many of the people who post on the message boards suffer from other diseases, as well as Cushing’s. These links help to provide some information about these diseases.

~A ~

Acanthosis nigricans
This Topic on the Message Boards.

Acromegaly
This Topic on the Message Boards.

Addison’s Disease
This Topic on the Message Boards.

Adrenoleukodystrophy
This Topic on the Message Boards.


~B ~

Barrett’s esophagus


~C ~

Carney Complex
This Topic on the Message Boards.
New Support Group for Carney Complex.

Central Serous Retinopathy
This Topic on the Message Boards.

Congenital Adrenal Hyperplasia (CAH)
This Topic on the Message Boards.

Conn’s Syndrome
This Topic on the Message Boards.

Craniopharyngioma
This Topic on the Message Boards.


~D ~

Diabetes insipidus
This Topic on the Message Boards.


~E ~

Ectopic ACTH Syndrome
This Topic on the Message Boards.

Empty Sella
This Topic on the Message Boards.


~F ~

Fibromyalgia
This Topic on the Message Boards.


~G ~

Gigantism
This Topic on the Message Boards.


~H ~

Hirsuitism
This Topic on the Message Boards.

Hyperprolactinemia
This Topic on the Message Boards.

Hyperthyroidism
This Topic on the Message Boards.

Hypoalderostonism
This Topic on the Message Boards.

Hypocalcemia
This Topic on the Message Boards

Hypopituitarism
This Topic on the Message Boards.

Hypothyroidism
This Topic on the Message Boards.


~I ~

Insulin Resistance
This Topic on the Message Boards.


~K ~

Kidney Disease
This Topic on the Message Boards.


~L ~

Lyme Disease
This Topic on the Message Boards.


~M ~

Madelung’s Disease
This Topic on the Message Boards.

Menopause
This Topic on the Message Boards.

MEN Type 1
This Topic on the Message Boards.

Myasthenia Gravis
This Topic on the Message Boards.


~N ~

Nelson’s Syndrome
This Topic on the Message Boards.


~O ~

Osteopenia
This Topic on the Message Boards.

Osteoporosis
This Topic on the Message Boards.


~P ~

Panhypopituitarism
This Topic on the Message Boards.

PCOS
This Topic on the Message Boards.

Perimenopause
This Topic on the Message Boards.

Pheochromocytoma
This Topic on the Message Boards.

Pituitary dwarfism
This Topic on the Message Boards.

Premature menopause
This Topic on the Message Boards.

Primary pigmented nodular adrenocortical disease (PPNAD)
This topic on the Message Boards

Prolactinoma
This Topic on the Message Boards.

Pseudo Cushing’s
This Topic on the Message Boards


~R ~

Rathke’s cleft cyst
This Topic on the Message Boards.

ROHHAD (Rapid-Onset Obesity With Hypothalamic Dysfunction, Hypoventilation, and Autonomic Dysregulation Presenting in Childhood)
This Topic on the Message Boards


~S ~

Sheehan’s Syndrome
This Topic on the Message Boards.

Stein-Leventhal Syndrome
This Topic on the Message Boards.


~T ~

Thymoma
This Topic on the Message Boards.

Thyroid Gland Disorders
This Topic on the Message Boards.

Turner’s Syndrome
This Topic on the Message Boards.


~V ~

Von Hippel-Lindau disease
This Topic on the Message Boards.


~Z ~

Zollinger-Ellison Syndrome

Is Diabetes in Cushing’s Syndrome a Consequence of Hypercortisolism?

Eur J Endocrinol. 2013 Nov 19. [Epub ahead of print]

Is Diabetes in Cushing syndrome only a consequence of hypercortisolism?

Source

C Giordano, Dipartimento di Medicina Interna e Specialistica (Di.Bi.Mi.S) Sezione di Endocrinologia e Malattie del Metabolismo, University of Palermo, Palermo, Italy.

Abstract

OBJECTIVE:

Diabetes mellitus (DM) is one of the most frequent complications of Cushing syndrome (CS). Aim of the study was to define the changes in insulin sensitivity and/or secretion in relation to glucose tolerance categories in newly diagnosed CS patients.

DESIGN:

Cross-sectional study on 140 patients with CS.

METHODS:

113 women (80 with pituitary disease and 33 with adrenal disease, aged 41.7±15.7 yr) and 27 men (19 with pituitary disease and 8 with adrenal disease, aged 38.1±20.01 yr) at diagnosis were divided according to glucose tolerance into normal glucose tolerance (CS/NGT), impaired fasting glucose and/or impaired glucose tolerance (CS/prediabetes) and diabetes (CS/DM).

RESULTS:

71 patients belonged to CS/NGT (49.3%), 26 (18.5%) to CS/prediabetes and 43 (30.8%) to CS/DM. Significant increasing trends in the prevalence of family history of diabetes (p<0.001), metabolic syndrome (p<0.001), age (p<0.001) and waist circumference (p=0.043) and decreasing trends in HOMAβ (p<0.001)and Oral Dispositional Index (DIo) (p<0.002) were observed among the groups. No significant trend in fasting insulin, AUC INS, ISI-Matsuda and VAI was detected.

CONCLUSIONS:

Impairment of glucose tolerance is characterized by the inability of β-cells to adequately compensate insulin resistance through increased insulin secretion. Age, genetic predisposition and lifestyle, in combination with duration and degree of hypercortisolism, strongly contribute to the impairment of glucose tolerance in the natural history of CS. A careful phenotypic evaluation of glucose tolerance defects in patients with CS proves useful for the identification of patients at high risk for metabolic complications.

PMID:
24255133
[PubMed – as supplied by publisher]
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