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1993.12.30 : Approval of Aprotinin

Food and Drug Administration
December 30, 1993

The Food and Drug Administration today announced approval of
aprotinin, a drug that can reduce the need for blood transfusions
in patients undergoing heart bypass surgery. 

In 1991, 265,000 coronary bypass graft operations were performed to
replace diseased blood vessels.  Excessive bleeding is a frequent
complication of this surgery.  

Two placebo-controlled clinical trials conducted in the United
States demonstrated that aprotinin effectively reduced blood loss
and decreased the need for transfusions.  In one study, 42 percent
of patients treated with aprotinin needed at least one unit of
blood, compared to 77 percent who did not receive the drug.  The
second study showed similar results.  

Aprotinin was studied for use mainly in heart surgery because the
circulation of the blood outside the body in this surgery increases
the likelihood of excessive bleeding during and after surgery.  Its
use should be reserved for high-risk patients, however, because
severe allergic reactions can result from using it more than once
in a patient.  Kidney toxicity was also a problem in some patients
in the trials.  

"Aprotinin can reduce the risks of bypass surgery for some
patients," said FDA Commissioner David A. Kessler, M.D.  "Fewer
transfusions mean a much lower risk of infection or possible
adverse reactions to the blood."  

Aprotinin will be most useful in patients at high risk of bleeding
-- particularly patients undergoing repeat bypass surgery or
patients with clotting defects, for example.  The drug may also be
used in patients with rare blood types or in other cases when
access to blood is limited.  

Miles Inc. of West Haven, Conn., will market aprotinin under the
name Trasylol Injection.  The company has agreed to conduct further
studies to evaluate the drug in other types of surgery associated
with a high risk of bleeding, such as organ transplants and aortic
reconstruction.  

FDA is one of the eight Public Health Service agencies in HHS.  
FOR IMMEDIATE RELEASE
Food and Drug Administration
Dec. 30, 1993
Monica Revelle (301) 443-4177

The Food and Drug Administration today announced the licensing of
the drug dornase alfa, commonly called DNase, as the first product
in 30 years specifically developed to treat cystic fibrosis, an
inherited disorder that affects about 30,000 Americans.  

DNase, a product of recombinant DNA technology, has been shown to
reduce the frequency of respiratory infections and improve lung
function in cystic fibrosis patients.  It was licensed nine months
after being submitted for review.  

"Although this new product is not a cure for cystic fibrosis, the
clinical data show that it can make a real difference in the
quality of life for many patients," said FDA Commissioner David A.
Kessler, M.D.  "This is the first treatment that specifically
improves lung function in cystic fibrosis patients."  

The drug was evaluated in a six-month, multi-center, placebo-
controlled clinical trial of 968 cystic fibrosis patients 5 years
of age and older.  Daily doses of DNase, when used in conjunction
with standard therapies, reduced the risk of severe respiratory
tract infections by 27 percent and increased patients' lung
function.

Side effects include inflammation of the throat, chest pain, voice
alteration and laryngitis.  

No clinical trials have been conducted to demonstrate safety and
effectiveness of DNase in children under 5, in patients with
breathing function measured at less than 40 percent or in patients
for longer than 12 months.  

In providing an expedited review, FDA followed procedures
established to implement the Prescription Drug User Fee Act of
1992, which provides additional resources to FDA to speed up review
of drugs and biologics submitted to the agency for approval.  

The application for DNase was jointly reviewed with the Canadian
Health Protection Branch and was approved simultaneously in both
countries.  

Cystic fibrosis is characterized by thick mucous secretions in the
lungs.  The retention of this mucous in the airways contributes to
reduced lung function and chronic lung infections.  Respiratory
complications are the major cause of death among patients.  

Due to the relatively low incidence of cystic fibrosis, FDA has
designated DNase an "orphan" product.  This designation provides
financial incentives for companies developing products for rare
diseases -- those affecting fewer than 200,000 people in the United
States.  

DNase is manufactured by Genetech Inc. of San Francisco, Calif.,
and is marketed under the trade name Pulmozyme.  

FDA is one of the eight Public Health Service agencies within HHS.