Answers
03/20/1990
Treatment IND Update
TREATMENT IND UPDATE
The following may be used to answer inquiries about FDA's "treatment
IND" program:
Under the FDA treatment IND (investigational new drug) regulations
enacted in l987, drugs that are in controlled clinical trials can be
provided outside these trials to treat patients with serious or immediately
life-threatening diseases for which no comparable or satisfactory alternate
therapy exists.
Certain safeguards must be observed, including requirements:
-- That the patient is fully informed of the risks and expressly
consents.
-- That the drug is not promoted or otherwise "commercialized," though
drug companies can charge patients to recover the cost of the drug's
manufacture, research, development and handling.
-- That clinical trials are underway and continue unimpeded, and the
sponsor of the drug actively pursues marketing approval of the drug with
"due diligence."
FDA has approved l8 drugs for use under its treatment IND program.
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The most recent approval, baclofen for infusion into the spinal canal
(intrathecal infusion) using the Medtronic SynchroMed Infusion System, was
approved for those multiple sclerosis and spinal cord injury patients with
severe and chronic spasticity who cannot tolerate or do not respond to oral
baclofen. The drug has been available in an oral form for treating
spasticity for l2 years. But about 20,000 patients who suffer painful
chronic spasticity either do not experience sufficient benefit from the oral
preparation or suffer unacceptable side effects.
One study has indicated that intrathecal baclofen can have a dramatic
clinical effect in some patients who had not responded to oral baclofen.
The SynchroMed Infusion System, an infusion pump containing baclofen, is
placed beneath the skin in the patient's abdomen. It can be programmed via
radio signals to dispense the drug through a small catheter inserted into
the spinal canal. The device is refilled every four to eight weeks by
injection with a hypodermic needle through the skin and a self-sealing
rubber cover on the pump.
Although there has been significant benefit from intrathecal baclofen in
some patients, overall exposure of patients is small to date, and there is
need for caution and careful patient monitoring. At least one death not
explained by any other cause has occurred with the intrathecal infusion of
baclofen. The informed consent obtained from patients in the treatment IND
will make note of this, and FDA will require that doctors using
the device call each patient under their care every week for at least the
first six months of treatment and that the treatment IND sponsor call each
doctor each week to ascertain the status of all patients.
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Physicians treating patients with severe spasticity who have failed oral
antispastic medications can contact Medtronic Inc. at 800-328-08l0 or
6l2-572-5000 for detailed information about enrollment in the treatment
protocol and the drug's distribution.
A list of previously approved treatment IND's and their status follows.
TREATMENT IND LIST
Drug: Cytomegalovirus immune globulin (CMV-IG)
Treatment IND Granted: October 1987
Indication: Prevention of cytomegalovirus infections in renal transplant
patients.
Sponsor: Comonwealth of Massachusetts, Department of Public Health.
Market Approval Date: Not yet approved.
Drug: Ifosfamide & Mesna
Treatment IND Granted: December 1987
Indication: Germ cell carcinoma.
Sponsor: National Cancer Institute.
Market Approval Date: December 31, 1988.
Drug: Trimetrexate
Treatment IND Granted: February 1988
Indication: AIDS patients with Pneumocystis carinii pneumonia who are
intolerant to standard forms of therapy.
Sponsor: National Institute of Allergy and Infectious Diseases.
Market Approval Date: Not yet approved.
Drug: Anafranil (clomipramine HCl)
Treatment IND Granted: June 1988
Indication: Severe cases of Obsessive Compulsive Disorder.
Sponsor: Ciba-Geigy.
Market Approval Date: December 29, 1989.
Drug: Eldepryl (selegiline HCl)
Treatment IND Granted: June 1988
Indication: Severe Parkinson's Disease.
Sponsor: Somerset Pharmaceuticals.
Market Approval Date: June 5, 1989.
Drug: Pentostatin
Treatment IND Granted: July 1988
Indication: Hairy cell leukemia refractory to alpha interferon.
Sponsor: National Cancer Institute.
Market Approval Date: Not yet approved.
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Drug: Teniposide
Treatment IND Granted: October 1988
Indication: Relapsed or refractory acute lymphoblastic leukemia.
Sponsor: National Cancer Institute.
Market Approval Date: Not yet approved.
Drug: Ganciclovir
Treatment IND Granted: November 1988
Indication: CMV retinitis in AIDS patients.
Sponsor: National Institute of Allergy and Infectious Diseases.
Market Approval Date: June 23, 1989.
Drug: Pentamidine Isethionate Aerosol
Treatment IND Granted: February 1989
Indication: Prevention of Pneumocystis carinii penumonia (PCP) in AIDS
patients who have recovered from an episode of PCP.
Sponsor: LyphoMed.
Market Approval Date: June 15, 1989.
Drug: Levamisole hydrochloride
Treatment IND Granted: May 1989
Indication: For use (with 5-fluorouracil) as an adjuvant treatment for
Dukes C adenocarcinoma of the colon.
Sponsor: National Cancer Institute.
Market Approval Date: Not yet approved.
Drug: Erythropoietin (EPO)
Treatment IND Granted: June 1989
Indication: Treatment of AZT related anemia in HIV positive patients.
Sponsor: Ortho.
Market Approval Date: Not yet approved.
Drug: Exosurf (synthetic pulmonary surfactant)
Treatment IND Granted: July 1989
Indication: Prophylactic treatment of newborns likely to develop
respiratory distress syndrome and rescue treatment of newborns with
confirmed RDS.
Sponsor: Burroughs Wellcome.
Market Approval Date: Not yet approved.
Drug: 2'3' dideoxyinosine (ddI)
Treatment IND Granted: September 1989
Indication: Treatment of AIDS patients intolerant to AZT.
Sponsor: Bristol Myers.
Market Approval Date: Not yet approved.
Drug: Survanta (bovine pulmonary surfactant)
Treatment IND Granted: October 1989
Indication: Prevention and treatment of respiratory distress syndrome in
premature infants.
Sponsor: Ross Labs.
Market Approval Date: Not yet approved.
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Drug: zidovudine
Treatment IND Granted: October 1989
Indication: Treatment of children under the age of 13 who have AIDS or are
suffering from symptoms of advanced infection with the AIDS virus.
Sponsor: Burroughs Wellcome.
Market Approval Date: Not yet approved.
Drug: mannose terminated beta-glucocerebrosidase
Treatment IND Granted: November 1989
Indication: Treatment of patients with chronic Gaucher's disease.
Sponsor: Genzyme Corporation.
Market Approval Date: Not yet approved.
Drug: fludarabine phosphate
Treatment IND Granted: November 1989
Indication: Chronic Lymphocytic Leukemia
Sponsor: National Cancer Institute
Market Approval Date: Not yet approved.