-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
▎The content team editor of WuXi AppTec recently announced that the U.
S.
FDA has accepted the company’s New Drug Application (NDA) for the selective RARγ agonist palovarotene for the treatment of a rare genetic disease with progressive muscular ossification (Fibrodysplasia ossificans progressiva, FOP).
The FDA also granted this NDA priority review qualification and is expected to respond before November 30 this year.
The press release stated that if approved, palovarotene will become the world's first therapy for FOP.
Another common name of FOP is "Stone Man Disease", which means that as the disease progresses, people will be unable to move like a stone.
The main symptom of this rare disease is that the soft and elastic muscles and connective tissues in the human body will become inflamed after minor injuries, and will turn into bones, locking the joints that could otherwise move freely in one place.
The locking of the elbow and knee joints will keep the patient's arms and legs fixed at an angle forever, the fusion of the jaw joints will affect their ability to eat and speak, and the ossification of the tissues around the ribs in the thoracic cavity will cause them to have difficulty breathing.
For patients with this rare disease, even with the most delicate protection, ossification will continue to occur, and eventually a second set of bones will grow in their bodies.
Their average life expectancy is only over 50 years.
Related reading: The disease gave her a second bone, and she turned it into a gift to the pharmaceutical industry.
Palovarotene is a selective RARγ inhibitor obtained by Ipsen's US$1.
3 billion acquisition of Clementia Pharmaceuticals.
It has been granted breakthrough therapy designation by the US FDA for the treatment of FOP.
NDA is mainly based on data from the ongoing MOVE clinical trial, which is FOP's first global multi-center phase 3 clinical trial.
The post-hoc analysis of the primary endpoint of the trial showed that compared with untreated patients in the natural history study, patients treated with palovarotene reduced the volume of new heterotopic ossification by an average of 62% each year (p=0.
0292).
Dr.
Howard Mayer, Executive Vice President and Head of Research and Development of Ipsen, said: “Because there are no approved treatments to treat this progressive and debilitating disease, there is still a large unmet medical need for FOP patients.
It has been 15 years since the mutation of ALK2/ACVR1 in the gene that causes FOP was discovered.
The submission of a new drug application for palovarotene means that the world's first potential therapy is expected to be born.
We will focus on working closely with regulatory agencies to provide FOP patients around the world Brings this potential treatment option.
" Reference: [1] Ipsen Confirms US FDA Accepts New Drug Application for Palovarotene as the First Potential Treatment Worldwide for Fibrodysplasia Ossificans Progressiva (FOP).
Retrieved May 31, 2021, from https://Note: This article aims to introduce the progress of medical and health research, and is not a treatment plan recommendation.
If you need guidance on the treatment plan, please go to a regular hospital for treatment.
S.
FDA has accepted the company’s New Drug Application (NDA) for the selective RARγ agonist palovarotene for the treatment of a rare genetic disease with progressive muscular ossification (Fibrodysplasia ossificans progressiva, FOP).
The FDA also granted this NDA priority review qualification and is expected to respond before November 30 this year.
The press release stated that if approved, palovarotene will become the world's first therapy for FOP.
Another common name of FOP is "Stone Man Disease", which means that as the disease progresses, people will be unable to move like a stone.
The main symptom of this rare disease is that the soft and elastic muscles and connective tissues in the human body will become inflamed after minor injuries, and will turn into bones, locking the joints that could otherwise move freely in one place.
The locking of the elbow and knee joints will keep the patient's arms and legs fixed at an angle forever, the fusion of the jaw joints will affect their ability to eat and speak, and the ossification of the tissues around the ribs in the thoracic cavity will cause them to have difficulty breathing.
For patients with this rare disease, even with the most delicate protection, ossification will continue to occur, and eventually a second set of bones will grow in their bodies.
Their average life expectancy is only over 50 years.
Related reading: The disease gave her a second bone, and she turned it into a gift to the pharmaceutical industry.
Palovarotene is a selective RARγ inhibitor obtained by Ipsen's US$1.
3 billion acquisition of Clementia Pharmaceuticals.
It has been granted breakthrough therapy designation by the US FDA for the treatment of FOP.
NDA is mainly based on data from the ongoing MOVE clinical trial, which is FOP's first global multi-center phase 3 clinical trial.
The post-hoc analysis of the primary endpoint of the trial showed that compared with untreated patients in the natural history study, patients treated with palovarotene reduced the volume of new heterotopic ossification by an average of 62% each year (p=0.
0292).
Dr.
Howard Mayer, Executive Vice President and Head of Research and Development of Ipsen, said: “Because there are no approved treatments to treat this progressive and debilitating disease, there is still a large unmet medical need for FOP patients.
It has been 15 years since the mutation of ALK2/ACVR1 in the gene that causes FOP was discovered.
The submission of a new drug application for palovarotene means that the world's first potential therapy is expected to be born.
We will focus on working closely with regulatory agencies to provide FOP patients around the world Brings this potential treatment option.
" Reference: [1] Ipsen Confirms US FDA Accepts New Drug Application for Palovarotene as the First Potential Treatment Worldwide for Fibrodysplasia Ossificans Progressiva (FOP).
Retrieved May 31, 2021, from https://Note: This article aims to introduce the progress of medical and health research, and is not a treatment plan recommendation.
If you need guidance on the treatment plan, please go to a regular hospital for treatment.
