Drug Safety Information for KUVAN (Sapropterin dihydrochloride)

FDA Safety-related Labeling Changes for KUVAN (SAPROPTERIN DIHYDROCHLORIDE) Rx Drug: Safety Information Link

Required post-approval safety study:

BioMarin commits to designing and implementing a registry of patients with PKU being treated with Kuvan (sapropterin dihydrochloride) that will be established to obtain longterm clinical status information. Information will be collected on patient demographics, specifics of treatment with Kuvan (sapropterin dihydrochloride), clinical status, neurocognitive assessments, growth and development (for patients who are pre-pubertal at the start of treatment), and adverse events. This registry will be designed so that detailed clinical status information is collected at registry entry and on a six- to twelve-month basis for at least 15 years. BioMarin commits to conducting one sub-study within the registry that will evaluate the effect of Kuvan (sapropterin dihydrochloride) on pregnancy and lactation. The registry data will be analyzed at yearly intervals and the results will be submitted in annual reports for IND 69,708. A registry protocol will be submitted to CDER by May 25, 2008, for concurrence, and the registry will be initiated by November 25, 2008. The final study report under this registry will be submitted to CDER by May 25, 2025. Due Date: 2025-05-25

Original FDA Drug Approval Date for KUVAN: 2007-12-13

Adverse Drug Reactions for KUVAN* (Sapropterin dihydrochloride)

These charts and graphs are based on reports received through the FDA Adverse Event Reporting System (FAERS) program (see below).

Top 20 Adverse Effects Associated with KUVAN
(reported in FDA Medwatch/FAERS Reports)

	Side Effect	# of FDA Reports
1	Drug ineffective	101
2	Headache	88
3	Vomiting	76
4	Diarrhoea	61
5	Abdominal pain upper*	58
6	Dizziness	51
7	Nausea	50
8	Pyrexia	39
9	Abdominal discomfort	37
10	Cough	30
11	Oropharyngeal pain	27
12	Decreased appetite	27
13	Amino acid level increased	27
14	Nasopharyngitis	26
15	Convulsion	26
16	Psychomotor hyperactivity	26
17	Abdominal pain	24
18	Fatigue	24
19	Anxiety	24
20	Malaise	23

* This side effect also appears in "Top 10 Side Effects of KUVAN " in the drug's Review Summary based on AskaPatient reviews.

Top 10 Reasons for Taking KUVAN
(associated with FDA Medwatch/FAERS Reports)

	Reason	# of FDA Reports
1	Phenylketonuria	2166
2	Product used for unknown indication	192
3	Tetrahydrobiopterin deficiency	38
4	Rabies	27
5	Neurotransmitter level altered	9
6	Off label use	6
7	Metabolic disorder	6
8	Nervous system disorder	5
9	Dementia alzheimer's type	5
10	Amino acid level increased	4

*Also a top-10 reason in AskaPatient Review Summary.

Types of Adverse Events for KUVAN

Total Reports Filed with FDA: 2524

Number of FDA Adverse Event Reports by Patient Age for KUVAN

Total Reports Filed with FDA: 2524*

* Reports for drugs with the same active ingredients have been aggregated in this analysis: Sapropterin dihydrochloride (Tetrahydrobiopterin, Bh4, Kuvan)

Charts are based on 2524 reports filed with the FDA between 2004 and June 2015.

Adverse Event Reports are submitted to the FDA by pharmaceutical companies (mandatory reporting is required by manufacturers, distributors, or importers), health care providers such as doctors (voluntary reporting), and by patients themselves (voluntary reporting). More Information on FDA MedWatch (FDA Safety Information and Adverse Event Reporting Program)Voluntary reporting takes place under the FDA's MedWatch program, where health care professionals and consumers submit reports to FDA when they find a problem with a drug, medical device, biologic, or other FDA-regulated product. An adverse event is any undesirable experience or extreme side effect associated with the use of a medical product. Adverse events include: death, life threatening event, hospitalization, disability or permanent damage, congenital anomaly or birth defect, medical product use requiring a surgical or other intervention, or other serious medical situation believed to be caused by the drug.

Historical Analytics of FDA Adverse Event Reports Provided by Druginformer.com. FDA Drug Safety Information, including safety-related labeling updates, required post-approval safety studies, risk evaluation management strategies (REMS), alerts, and recalls are compiled by AskaPatient.com from the U.S. Food and Drug Administration.