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Ganaxolone (Epilepsy) – Forecast and Market Analysis to 2022

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Ganaxolone (Epilepsy) – Forecast and Market Analysis to 2022 Ganaxolone (Epilepsy) – Forecast and Market Analysis to 2022 Reference Code: GDHC1071DFR Publication Date: February 2013 Executive Summary Epilepsy: Key Metrics in the Epilepsy Markets The below figure illustrates ganaxolone sales for the US 2022 Market Sales and 5EU during the forecast period. US $43.17m Sales for Ganaxolone by Region, 2022 5EU $4.64m Total $47.81m 10% 2012 Total: $47.81m Key Events (2012–2022) Level of Impact Launch of ganaxolone in the US in 2019 ↑↑↑ US Launch of ganaxolone in the 5EU in 2019 ↑↑↑ 5EU Source: GlobalData Sales for Ganaxolone in the Epilepsy Market GlobalData expects Marinus Pharmaceuticals to launch 90% ganaxolone in the US and EU in 2019. We estimate that 2022 sales of ganaxolone will reach $47.81m across Source: GlobalData these markets. Key factors affecting the uptake of ganaxolone will include: What Do the Physicians Think? Novel mechanism of action and good tolerability Overall physicians expressed a need for more AEDs profile and favorable opinions of those in pipeline Efficacy profile is not significantly different from that development. of other marketed anti-epileptic drugs (AEDs) “Among intractable epilepsy patients, any drug that helps Ganaxolone is still in early development; possible treat an additional segment of them will be used, and lack of funding to conduct Phase III trials necessary because we don’t have a basis for using one or another, for commercialization if it’s attractive, it will be used more.” [US] key opinion leader, November 2012 Heavy competition in the market “Brivaracetam is an interesting concept because it’s supposed to be “Super Keppra,” the follow-on from Keppra. The Phase II studies were very promising, but I think the Phase III were a bit of a disappointment; there might be some methodological issues in terms of some patient selection issues that they have come across. But I think that’s one of the more interesting of the new drugs that I’m really wanting to see in clinical practice, particularly if, as the Phase II studies suggested, that some patients who haven’t responded to levetiracetam © GlobalData. This report is a licensed product and is not to be copied, reproduced, shared or resold in any form. Page 2 GDHC1071DFR / Published FEB 2013 Executive Summary are responders to this drug. So, I think that that’s going to “Do we need the sixth or seventh sodium blocker? Do we be one drug to watch.” need the tenth calcium channel modulator? I think we need new drugs, but more than that, I think we need new [EU] key opinion leader, November 2012 classes of drugs which address things differently.” However, with more than 20 existing AEDs, new [EU] key opinion leader, October 2012 ones currently entering the market, and more to come in the future, physicians are unsure of how all “If you ask me, my wish list would be disease-modifying these drugs will fit in the treatment landscape, and drugs that you could use once or twice, once you lack a way to differentiate between them, particularly determine that the person has a tendency for unprovoked in terms of efficacy. Also, there are no predictive seizures, and that would stop the process.” tools that would guide the choice of treatment from [EU] key opinion leader, November 2012 patient to patient. Physicians believe that a better understanding of the “We have about 25 AEDs for focal (partial) epilepsy, but disease mechanisms in epilepsy will be crucial to which one to choose? Upfront, it’s tough to say because developing more effective treatments. we don’t have any tests to say, ‘this is the lamotrigine “I would not discourage the development of drugs, but I patient and this is the levetiracetam patient’.” think we need to change the paradigm. But tochange the [EU] key opinion leader, October 2012 paradigm, we need to understand things better, so we In the future, physicians would like to see distinct need a lot of basic research.” new classes of AEDs that target different [EU] key opinion leader, November 2012 mechanisms, rather than more of the same drugs that currently dominate the market. © GlobalData. This report is a licensed product and is not to be copied, reproduced, shared or resold in any form. Page 3 GDHC1071DFR / Published FEB 2013 Table of Contents 1 Table of Contents 1 Table of Contents ............................................................................................................... 4 1.1 List of Tables ............................................................................................................... 7 1.2 List of Figures ............................................................................................................. 8 2 Introduction ......................................................................................................................... 9 2.1 Catalyst ....................................................................................................................... 9 2.2 Related Reports ........................................................................................................ 10 3 Disease Overview ............................................................................................................. 12 3.1 Classification of Seizures........................................................................................... 14 3.2 Etiology and Pathophysiology .................................................................................... 15 3.2.1 Etiology ............................................................................................................... 15 3.2.2 Pathophysiology.................................................................................................. 18 3.2.3 Prognosis ............................................................................................................ 23 3.2.4 Quality of Life ...................................................................................................... 24 4 Disease Management ....................................................................................................... 25 4.1 Diagnosis .................................................................................................................. 25 4.2 Treatment Guidelines ................................................................................................ 26 4.3 Clinical Practice ......................................................................................................... 27 5 Competitive Assessment ................................................................................................... 33 5.1 Overview ................................................................................................................... 33 5.2 Strategic Competitor Assessment .............................................................................. 34 6 Opportunity and Unmet Need ............................................................................................ 38 6.1 Overview ................................................................................................................... 38 6.2 Unmet Needs ............................................................................................................ 39 6.2.1 Refractory Epilepsy ............................................................................................. 39 6.2.2 Safety/Side Effect Profiles ................................................................................... 39 6.2.3 Curative/Disease-Modifying Agents ..................................................................... 40 6.2.4 Predictive Tools and Need for More Directed Treatments .................................... 41 © GlobalData. This report is a licensed product and is not to be copied, reproduced, shared or resold in any form. Page 4 GDHC1071DFR / Published FEB 2013 Table of Contents 6.2.5 Improved Preclinical Models and Clinical Trial Paradigms ................................... 42 6.2.6 Treatment Gap .................................................................................................... 43 6.3 Unmet Needs Gap Analysis ....................................................................................... 43 6.4 Opportunities ............................................................................................................. 45 6.4.1 Disease-Modifying Agents ................................................................................... 45 6.4.2 Improved Preclinical Models and Clinical Trial Paradigms ................................... 45 6.4.3 Treatment Gap .................................................................................................... 45 7 Pipeline Assessment......................................................................................................... 46 7.1 Overview ................................................................................................................... 46 7.2 Strategic Pipeline Assessment .................................................................................. 46 7.3 Pipeline by Phases of Development .......................................................................... 47 7.3.1 Phase III Pipeline ................................................................................................ 47 7.3.2 Phase III Pipeline – Reformulations/New Delivery Systems ................................. 47 7.3.3 Phase IIb Pipeline ............................................................................................... 48 7.3.4 Phase II Pipeline ................................................................................................
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