Health Medical Homework Help

As we have discussed, and as you have read during Module 2 of the course, Duchenne Muscular Dystrophy (DMD) is an X-linked heritable neuromuscular disorder with a prevalence in the US population of 1 in every 7,250 males aged 5-24 years.1 The disease has traditionally been characterized by a wide diversity of mutations at the Xp21 genetic locus, many of which result in DMD patients being unable to produce adequate levels of wild type dystrophin protein. The absence of dystrophin results over time in the deterioration of various muscle types, leading to the loss of ambulation, loss of respiratory and cardiac function, and ultimately death, typically by the time DMD patients reach their late twenties.

During Module 2, we have reviewed therapies introduced by two manufacturers to treat patients afflicted with DMD: Sarepta Therapeutics and PTC Therapeutics. Both have received approval from either the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) to commercialize at least one therapy for clinical utilization in DMD patients. There are other pharmaceutical and biologic product manufacturers that are developing new clinical treatments for DMD patients. In this Quiz, you are asked to focus on compound(s) currently in development for treatment of DMD by one of two manufacturers: Catabasis Pharmaceuticals or Wave Life Sciences. In answering the questions below, please choose a compound currently in development for treatment of DMD by ONE of these companies.

Each student is to work independently on this Quiz and is responsible for their own Quiz submission. Please answer these questions at the level you would answer if you were sending the information to FDA in a Briefing Book (for example for either a Type B Meeting or for an FDA Advisory Committee Meeting). Please cite your references when answering these questions.

Questions

1) What is the primary pharmacodynamic mechanism of action of the lead compound you have chosen? How do the pharmacodynamics of your chosen compound relate to the natural history of Duchenne Muscular Dystrophy disease progression and what impact does the compound have on progression of the disease? How do we know? Is the purported pharmacodynamic profile supported by non-clinical and/or clinical evidence?

2) What stage of development is the lead compound at from a regulatory perspective (assume that the manufacturer is seeking approval to market their DMD treatment in the US market)? Has the manufacturer received any expedited approval or other regulatory designation(s) from FDA?

3) What is the proposed indication(s) for use for the lead compound? How does this proposed indication(s) for use compare with the FDA approved labels for Sarepta Therapeutics’ DMD treatment?

4) What clinical studies are currently being conducted to support FDA marketing application(s) for either Catabasis Pharmaceuticals’ or Wave Life Sciences’ products?

5) Is the use of a genetic test and/or companion diagnostic required for inclusion in any pre-approval studies currently underway for either Catabasis Pharmaceuticals’ or Wave Life Sciences’ products?

6) If you were the Vice President of Regulatory Affairs at either Catabasis Pharmaceuticals or Wave Life Sciences how would you propose to FDA that the use of real-world evidence be incorporated into the regulatory decision-making process for marketing?