FDA Approves First Drug to Treat Growth Failure in Children With Prader-Willi Syndrome

- Additional Data Demonstrates Decrease in Fat Mass and Increase in

Lean Body Mass -



Jun 21, 2000, 01:00 ET from Pharmacia Corporation

    PEAPACK, N.J., June 21 /PRNewswire/ -- Pharmacia Corporation (NYSE:   PHA)
 announced today that the U.S. Food & Drug Administration (FDA) has approved
 and granted orphan drug status for GENOTROPIN(R) (somatropin [rDNA origin] for
 injection) in the long-term treatment of growth failure in children with
 Prader-Willi syndrome (PWS), a rare genetic disorder that causes short
 stature, an involuntary, continuous urge to eat, low muscle tone, and
 cognitive disabilities.
     It is estimated that 17,000 to 22,000 Americans have PWS.  Although a rare
 disorder, PWS is one of the 10 most common conditions seen in genetics clinics
 and the most common genetic cause of obesity that has been identified.(1)
     "Learning that their child has PWS can be a devastating discovery for
 parents.  Beyond the struggles to help their child cope with the physical and
 emotional problems inherent with the disorder, they also face a lifelong
 battle to control obesity potentially so severe it can threaten their child's
 life," said Janalee Heinemann, Executive Director, Prader-Willi Syndrome
 Association and parent of a child with PWS.  "After so many years with no
 officially recognized treatment for this disorder, the approval of GENOTROPIN
 for the syndrome represents a significant medical advance for children with
 PWS."
     Children with PWS are born with a dysfunction of the hypothalamus, the
 part of the brain that controls growth, pubertal development and feelings of
 hunger and satiety (fullness).  As a result, growth failure often occurs,
 accompanied by an uncontrollable urge to eat continuously.  This problem is
 compounded by the fact that people with PWS actually require considerably
 fewer calories to maintain an appropriate weight.  The combination of the
 constant urge to eat and a reduced need for calories results in the disorder's
 major medical concern -- potentially life-threatening obesity.
     "Individuals with Prader-Willi syndrome suffer from a complex genetic
 condition characterized by an abnormal appearance, short stature and obesity,"
 said Dr. Phillip Lee, Director of Endocrinology and Metabolism at Children's
 Hospital of Orange County, California.  "Despite having an insatiable
 appetite, individuals with PWS have markedly decreased amount of muscle, very
 low muscle strength and weak bones.  These characteristics can lead to severe
 medical complications and lifestyle limitations.  Growth hormone treatment of
 children with PWS improves growth, increases muscle mass, and can help to
 reduce body fat.  In my experience, these changes can result in a nearly
 normal body appearance and a greatly improved quality of life for these
 children and their families.  Growth hormone treatment gives new hope for a
 condition which has so far had few, if any, effective treatment options."
     In two randomized, open-label, controlled clinical trials, 23 children
 with PWS received GENOTROPIN.  A control group received standard care.  At the
 end of the first year of treatment, there was a clinically and statistically
 significant difference in the mean change in height between the treatment and
 control groups in both studies.  Linear growth continued in the second year
 when both groups received treatment with GENOTROPIN.  In addition, GENOTROPIN
 produced beneficial effects on body composition at one year, with an increase
 in lean body mass and decrease in fat mass.  There was no apparent impact on
 body weight.  The diagnosis of PWS should be confirmed by appropriate genetic
 testing.  Unlike the growth failure in growth hormone deficiency, the growth
 failure in PWS does not require confirmation with a growth hormone stimulation
 test.
     In these studies the following drug-related events were reported: edema,
 aggressiveness, joint pain, benign intracranial hypertension, hair loss,
 headache and muscular pain.  Scoliosis is commonly seen in patients with PWS.
 Physicians should be alert to this abnormality, which may manifest or progress
 during growth hormone therapy.  People with diabetes mellitus (type 1 or type
 2) or glucose intolerance should be monitored closely during treatment with
 GENOTROPIN because the insulin dosage may need to be adjusted.
     In the United States, GENOTROPIN Lyophilized Powder (somatropin [rDNA
 origin] for injection) has been approved for the long-term treatment of
 children with growth hormone deficiency (GHD) since August 1995 and of adults
 with GHD of either childhood or adult onset since November 1997.  Today,
 GENOTROPIN is the leading brand of growth hormone in the world and is the only
 growth hormone in the United States with an indication for PWS.  GENOTROPIN is
 available by prescription only.  Given the small estimated number of people
 with PWS, GENOTROPIN has been granted orphan drug status by the FDA for the
 PWS indication.  This designation is only given to treatments for which the
 potential patient population is under 200,000. This status entitles Pharmacia
 to seven years exclusivity to market the drug for PWS.
     Pharmacia Corporation is a leading global pharmaceutical company created
 through the merger of Pharmacia & Upjohn and Monsanto Company with its G.D.
 Searle & Co. unit. Pharmacia (NYSE:   PHA) has a broad product portfolio, a
 robust pipeline of new drugs, and an annual investment of more than $2 billion
 in pharmaceutical research and development.
     Healthcare professionals who have clinical questions about GENOTROPIN can
 call 1-800-253-8600, extension 3-8244.  Consumers who have questions about
 GENOTROPIN can call 1-888-691-6813.  Healthcare professionals and consumers
 can also visit the website at http://www.genotropin.com or e-mail us at
 genotropin@am.pnu.com.  Additional information may also be obtained by
 contacting the Prader-Willi Syndrome Association (http://www.pwsausa.org) at
 800-926-4797.
 
     GENOTROPIN is a registered trademark of Pharmacia Corporation.
 
     Pharmacia Corporation Website: http://www.pharmacia.com
 
     (1)  Prader-Willi Syndrome Association, 2000
 
 

SOURCE Pharmacia Corporation
    PEAPACK, N.J., June 21 /PRNewswire/ -- Pharmacia Corporation (NYSE:   PHA)
 announced today that the U.S. Food & Drug Administration (FDA) has approved
 and granted orphan drug status for GENOTROPIN(R) (somatropin [rDNA origin] for
 injection) in the long-term treatment of growth failure in children with
 Prader-Willi syndrome (PWS), a rare genetic disorder that causes short
 stature, an involuntary, continuous urge to eat, low muscle tone, and
 cognitive disabilities.
     It is estimated that 17,000 to 22,000 Americans have PWS.  Although a rare
 disorder, PWS is one of the 10 most common conditions seen in genetics clinics
 and the most common genetic cause of obesity that has been identified.(1)
     "Learning that their child has PWS can be a devastating discovery for
 parents.  Beyond the struggles to help their child cope with the physical and
 emotional problems inherent with the disorder, they also face a lifelong
 battle to control obesity potentially so severe it can threaten their child's
 life," said Janalee Heinemann, Executive Director, Prader-Willi Syndrome
 Association and parent of a child with PWS.  "After so many years with no
 officially recognized treatment for this disorder, the approval of GENOTROPIN
 for the syndrome represents a significant medical advance for children with
 PWS."
     Children with PWS are born with a dysfunction of the hypothalamus, the
 part of the brain that controls growth, pubertal development and feelings of
 hunger and satiety (fullness).  As a result, growth failure often occurs,
 accompanied by an uncontrollable urge to eat continuously.  This problem is
 compounded by the fact that people with PWS actually require considerably
 fewer calories to maintain an appropriate weight.  The combination of the
 constant urge to eat and a reduced need for calories results in the disorder's
 major medical concern -- potentially life-threatening obesity.
     "Individuals with Prader-Willi syndrome suffer from a complex genetic
 condition characterized by an abnormal appearance, short stature and obesity,"
 said Dr. Phillip Lee, Director of Endocrinology and Metabolism at Children's
 Hospital of Orange County, California.  "Despite having an insatiable
 appetite, individuals with PWS have markedly decreased amount of muscle, very
 low muscle strength and weak bones.  These characteristics can lead to severe
 medical complications and lifestyle limitations.  Growth hormone treatment of
 children with PWS improves growth, increases muscle mass, and can help to
 reduce body fat.  In my experience, these changes can result in a nearly
 normal body appearance and a greatly improved quality of life for these
 children and their families.  Growth hormone treatment gives new hope for a
 condition which has so far had few, if any, effective treatment options."
     In two randomized, open-label, controlled clinical trials, 23 children
 with PWS received GENOTROPIN.  A control group received standard care.  At the
 end of the first year of treatment, there was a clinically and statistically
 significant difference in the mean change in height between the treatment and
 control groups in both studies.  Linear growth continued in the second year
 when both groups received treatment with GENOTROPIN.  In addition, GENOTROPIN
 produced beneficial effects on body composition at one year, with an increase
 in lean body mass and decrease in fat mass.  There was no apparent impact on
 body weight.  The diagnosis of PWS should be confirmed by appropriate genetic
 testing.  Unlike the growth failure in growth hormone deficiency, the growth
 failure in PWS does not require confirmation with a growth hormone stimulation
 test.
     In these studies the following drug-related events were reported: edema,
 aggressiveness, joint pain, benign intracranial hypertension, hair loss,
 headache and muscular pain.  Scoliosis is commonly seen in patients with PWS.
 Physicians should be alert to this abnormality, which may manifest or progress
 during growth hormone therapy.  People with diabetes mellitus (type 1 or type
 2) or glucose intolerance should be monitored closely during treatment with
 GENOTROPIN because the insulin dosage may need to be adjusted.
     In the United States, GENOTROPIN Lyophilized Powder (somatropin [rDNA
 origin] for injection) has been approved for the long-term treatment of
 children with growth hormone deficiency (GHD) since August 1995 and of adults
 with GHD of either childhood or adult onset since November 1997.  Today,
 GENOTROPIN is the leading brand of growth hormone in the world and is the only
 growth hormone in the United States with an indication for PWS.  GENOTROPIN is
 available by prescription only.  Given the small estimated number of people
 with PWS, GENOTROPIN has been granted orphan drug status by the FDA for the
 PWS indication.  This designation is only given to treatments for which the
 potential patient population is under 200,000. This status entitles Pharmacia
 to seven years exclusivity to market the drug for PWS.
     Pharmacia Corporation is a leading global pharmaceutical company created
 through the merger of Pharmacia & Upjohn and Monsanto Company with its G.D.
 Searle & Co. unit. Pharmacia (NYSE:   PHA) has a broad product portfolio, a
 robust pipeline of new drugs, and an annual investment of more than $2 billion
 in pharmaceutical research and development.
     Healthcare professionals who have clinical questions about GENOTROPIN can
 call 1-800-253-8600, extension 3-8244.  Consumers who have questions about
 GENOTROPIN can call 1-888-691-6813.  Healthcare professionals and consumers
 can also visit the website at http://www.genotropin.com or e-mail us at
 genotropin@am.pnu.com.  Additional information may also be obtained by
 contacting the Prader-Willi Syndrome Association (http://www.pwsausa.org) at
 800-926-4797.
 
     GENOTROPIN is a registered trademark of Pharmacia Corporation.
 
     Pharmacia Corporation Website: http://www.pharmacia.com
 
     (1)  Prader-Willi Syndrome Association, 2000
 
 SOURCE  Pharmacia Corporation