PHASE III CLINICAL TRIALS

Phase III studies aim to be the definitive assessment of the drug under investigation, providing additional safety data, confirming efficacy, and providing the necessary comparative data where an approved therapy is available. These studies are randomized, controlled, multicenter trials enrolling large numbers of patients. The actual number of patients studied can be as large as 3,000 to 5,000, but is also dependent on the number of patients who suffer from the target disease or condition.

Once the comprehensive Phase III studies have been completed, all available evidence, including all preclinical and clinical data collected before and during the clinical trials, is compiled to form the registration dossier. This dossier (e.g., the New Drug Application [NDA] in the US, or the Marketing Authorisation Application [MAA] in the EU) is then submitted to the relevant regulatory authorities in order to obtain approval to market the drug in the respective jurisdictions.

If the regulatory authorities are satisfied that the information provided in the dossier shows that the drug has fulfilled the relevant safety and efficacy criteria, approval to market the drug is granted.

ACTELION'S PHASE III PORTFOLIO:

Compound Indication Study Status

Cadazolid

 

Clostridium difficile- associated diarrhea

IMPACT

 

Ongoing 

Macitentan Pediatric PAH TOMORROW  Initiating
Macitentan Portopulmonary hypertension (PoPH) PORTICO Ongoing
Macitentan Fontan-palliated RUBATO Initiating
Ponesimod Multiple sclerosis OPTIMUM Ongoing
Ponesimod Multiple sclerosis POINT Ongoing
 

Administrative Assistant, US 

 

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