Pipeline

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M. DMD medicine

Rare genetic diseases in children
evelopment of medicine for Duchenne Muscular Dystrophy (DMD)
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    Efficacy Test

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    Toxicity Test

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    Clinical Test

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Source : https://auroshealthcare.wordpress.com/

Characteristics of the disease

  • 80 to 85% of all patients with muscular dystrophy are DMD patients
  • Ratio of nearly 1 in 3,500 newborn boys
  • Symptoms such as posture deformation which typically begin at the age of 3-4, leading to gradual atrophy of muscles
    → Most die in their early 20s when respiratory muscles become too weak
  • Currently, a full recovery is not possible due to the absence of medicine, which increases the necessity of developing a new drug

Selected Myostatin, an inhibitor of muscle creation, as the target antigen

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Global market value

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Status

Approved with the Orphan Drug Designation (ODD) by the U.S. FDA in February 2018
→ Increased possibility for acquisition of PRV

Orphan Drug Designation

  • Provision of research subsidies during the clinical test period
  • Tax deductions for clinical test expenses
  • Cost exemption for the review of new drug approval applications
  • Permission of conditional sales after Clinical Phase 2
  • Assignment of 7-year, exclusive marketing rights after permission is granted for marketing
  • [SAMSUNG MEDICAL CENTER] Signed an agreement for joint R&D in January 2012
  • Selected for and executed the assignment to develop advanced medical technology given by Ministry of Health and Welfare (2015 - 2018)

Strategy

  • Complete Clinical Phase 2 · Acquire permission for marketing from U.S. FDA
  • Secure additional technical partners → Establish a joint venture with a local partner or proceed with licensing out
  • Acquire PRV
  • A voucher received when developing an innovative new drug for rare diseases designated by the U.S. FDA : The period for approval of the development company's other medicines reduced to 6 months
    → Period of revenue may be extended, which leads to increases in sales
  • Possible to use or sell directly : The current sales value of PRV sits at over KRW 210 billion on average

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  • Status of PRV acquisition in relation to DMD disease
Year Company Drug Comments
2017 Emflaza
(deflazacort)		 FDA Press
release
2016 Exondys51
(eteplirsen)
Sarepta has sold PRV
to Gilead for $125m

Source : http://priorityreviewvoucher.org