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Phase 2 Study of ENB-0040 for the Treatment of Hypophosphatasia

Award Number: R01FD003745
ORGANIZATION: FOOD AND DRUG ADMINISTRATION
OPDIV: FDA
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 06/20/2011
PERIOD OF PERFORMANCE END DATE: 05/31/2014

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Phase 2 Study of ENB-0040 for the Treatment of Hypophosphatasia - DESCRIPTION (provided by applicant): Hypophosphatasia is a rare inherited disease due to deficiency of tissue non-specific alkaline phosphatase, an enzyme essential for the proper formation of bones. Hypophosphatasia appears in various forms largely depending upon the age of the patient. The most severe form is diagnosed before birth and is invariably lethal. If diagnosed after birth or later in life, the survival rate improves. Even though the genetic defect is known, presently there is no effective treatment for this disease. Enobia Pharma has developed ENB-0040, a fusion protein composed of human recombinant tissue non-specific alkaline phosphatase, for the treatment of hypophosphatasia. The objective of the present clinical study is to test the long-term safety and effectiveness of ENB-0040 to treat infant stage hypophosphatasia. All of the patients in this extension study must have completed a prior clinical study of ENB-0040. The duration of treatment with ENB-0040 in the extension study will be one year. During that year, eligible infants with hypophosphatasia will receive the drug three times per week by injection under the skin. Small quantities of blood and urine will be taken at intervals to follow the progress of the treatment and to monitor for any problems. X-rays will also be used at various times to assess the progress of bone formation.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2014 ( Subtotal = $0 )
2014	2013	ALEXION PHARMA INTERNATIONAL SARL	LAUSANNE	34 AVENUE DU TRIBUNAL-FEDERAL		1005		CHE	Food and Drug Administration Research	002	3	9/4/2014	NON-COMPETING CONTINUATION	$0
2014	2013	ALEXION PHARMA INTERNATIONAL SARL	LAUSANNE	34 AVENUE DU TRIBUNAL-FEDERAL		1005		CHE	Food and Drug Administration Research	003	2	9/4/2014	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$0
2014	2013	ALEXION PHARMA INTERNATIONAL SARL	LAUSANNE	34 AVENUE DU TRIBUNAL-FEDERAL		1005		CHE	Food and Drug Administration Research	001	3	6/10/2014	NON-COMPETING CONTINUATION	$0
2014	2013	ALEXION PHARMA INTERNATIONAL SARL	LAUSANNE	34 AVENUE DU TRIBUNAL-FEDERAL		1005		CHE	Food and Drug Administration Research	000	3	1/13/2014	NON-COMPETING CONTINUATION	$0
2014	2011	ALEXION PHARMA INTERNATIONAL SARL	LAUSANNE	34 AVENUE DU TRIBUNAL-FEDERAL		1005		CHE	Food and Drug Administration Research	004	1	9/9/2014	NEW	$0
														Subtotal = $0

Issue Date FY: 2013 ( Subtotal = $800,000 )
2013	2013	ALEXION PHARMACEUTICALS, INC	352 KNOTTER DRIVE	CHESHIRE	CT	06410	CAPITOL	USA	Food and Drug Administration Research	002	3	7/8/2013	NON-COMPETING CONTINUATION	$400,000
2013	2013	ALEXION PHARMACEUTICALS, INC	352 KNOTTER DRIVE	CHESHIRE	CT	06410	CAPITOL	USA	Food and Drug Administration Research	001	2	3/21/2013	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$400,000
2013	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL		H1W4A		CAN	Food and Drug Administration Research	000	1	3/19/2013	NEW	$0
														Subtotal = $800,000

Issue Date FY: 2012 ( Subtotal = $0 )
2012	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL		H1W4A		CAN	Food and Drug Administration Research	000	1	11/22/2011	NEW	$0
2012	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL		H1W4A		CAN	Food and Drug Administration Research	002	1	9/25/2012	NEW	$0
2012	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL		H1W4A		CAN	Food and Drug Administration Research	001	1	5/24/2012	NEW	$0
														Subtotal = $0

Issue Date FY: 2011 ( Subtotal = $400,000 )
2011	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL		H1W4A		CAN	Food and Drug Administration Research	001	1	9/8/2011	NEW	$0
2011	2011	ENOBIA PHARMA INC	2901 RACHEL STREET EAST	MONTREAL				CAN	Food and Drug Administration Research	000	1	6/21/2011	NEW	$400,000
														Subtotal = $400,000

Grand Total All Awards = $1,200,000

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Phase 2 Study of ENB-0040 for the Treatment of Hypophosphatasia

Award Number: R01FD003745

ORGANIZATION: FOOD AND DRUG ADMINISTRATION

OPDIV: FDA

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 06/20/2011

PERIOD OF PERFORMANCE END DATE: 05/31/2014

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