FDA BRIEF, Week of February 6, 2017

FDA approved


EMFLAZA (deflazacort) tablets, oral suspension

Marathon Pharmaceuticals, Northbrook, IL, USA

Image result for emflaza
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INDICATION: Treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older.

UNMET NEED:

  • DMD occurs in about 1/3,600 male infants worldwide
  • Caused by absence of dystrophin; symptoms between 3 -5 trs of age
  • Lose ability to perform activities independently; life-threatening heart and respiratory conditions can occur

REG PATHWAY: NDA

  • Fast Track designation, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Priority Review Voucher
  • marketed outside US

MECHANISM OF ACTION:  Corticosteroid prodrug, acts through glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects; precise mechanism unknown

EFFICACY:

  • Multicenter, randomized, double-blind, placebo-controlled, 52-week study, n=196 male pediatric patients, documented dystrophin gene mutation, EMFLAZA vs placebo at 12 weeks; vs active comparator at additional 40 weeks
  • Primary Endpoint: Change in Baseline and Week 12 in average strength of 18 muscle groups using MRC 11-point scale
  • Significantly greater vs placebo (p=0.017) at week 12
  • Persistence of the treatment effect at week 52
  • Additional randomized, double-blind, placebo-controlled, 104-week vs placebo, n=29 (6-12 yr)
  • Average muscle strength score at 2 years not statistically significant; patients on EMFLAZA appeared lose the walking ability later than placebo

SAFETY:

  • Side effects similar to other corticosteroids
  • Most common : Facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism) and excessive fat around the stomach (central obesity)

LABEL


GUIDANCE: Dear Healthcare provider Letters

HCP.JPG

OVERVIEW:

Dear Health Care Provider (DHCP) letters intended to alert physicians and other health care providers about important new or updated information on  human drug or biologic

  • Often in the form of a mass mailing from the manufacturer
  • Need to improve effectiveness in communicating drug information

SCOPE:

  • FDA consultation on development
  • When to use and which type to use
  • Important Drug Warning Letters
  • Important Prescribing Information Letters
  • Important Correction of Drug Information Letters
  • Content and Format
  • Assessment of impact

READ


 

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