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Gaus Pharma

Course: RGA 6201

Instructor: Dr. Anupama Gaur

Gaus Pharma Overview

Hypothyroidism

Market Scenario

Gausroid

Regulatory plan

Post approval strategy

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“Gaus” means “Foolish Person”.

US based Pharmaceutical Company

Located in Boston

700 Employees

40 Veteran Regulatory Employees.

Filing NDA for Gausroid

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Deficiency of Thyroid

Hormone.

Acquired or Congenital.

2-5% World Population

3.7% in U.S Population

(~Ten million).

Women > Men

More risk in Elders.

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Levothyroxine Sodium e.g.

*Synthroid(Abbott)-$520 million

*Levoxyl (Pfizer)- $15.4 million

Problems:

-Effect on bone density

-Unstable

-Potency problem

-Costly

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Levothyroxine Calcium.

Oral Replacement Therapy

New Improved Formulation:

-Restores Bone Mineral Density

-Lesser Adverse Event

-More Stable

-More Potent

-Economical

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IND Application Filed in eCTD

NDA Application Filed in eCTD

Label Expansion Strategies

GAUSROID

DEVELOPMENT

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Pharmacology

Toxicology

Single-dose toxicity

Repeat –dose toxicity

Genotoxicity

Carcinogenicity

Immunotoxicity

Reproductive and developmental toxicity

• Primary Pharmacodynamics

• Secondary Pharmacodynamics

• Safety Pharmacology

Pharmacodynamics

•Absorption,

•Distribution,

•Metabolism

• Excretion

Pharmacokinetics

*CFR Title 21 part 58 (GLP)

*ICH Guidelines: Safety, Quality, MultidisciplinaryNon

Clinical studies

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Gausroid CMC Complies with:

CFR Title 21 part 211 (cGMP)

ICH Guidelines

CMC Contains:

Drug Substance and Drug Product, ICH Q6A

Stability, ICH Q1A (R2)

Manufacturing Process Development

Quality, Identity and Purity ICH Q6A

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Pre IND:

-Type B Meeting as per 21 CFR 312.82.

Purpose:

To Discuss Scope and Design of Phase I.

To Investigate Shortcomings.

eCTD Format through FDA’s Electronic

Submission Gateway.

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Clinical Trials After IND Approval

Three phases Studies

Multi Centers Clinical Trials in US

Randomized, Double-Blinded,

Placebo Concurrent trials.

Conducted on Adults

Age 21 yrs-75 yrs.

ICH E6 guidelines: GCP

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Clinical Studies

Conducted on 50 Healthy Volunteers

Purpose: To Ensure Safety

*To Evaluate Pharmacological Action.

*To Determine Side EffectsConducted on 600 Adult Patients

Purpose: To Start Efficacy Studies and

selecting Dose for Phase III

*To Evaluate Effectiveness for Proposed

Indication

*Short Term Side Effects and RisksOn 2500 Patients with 40 weeks follow up

Purpose: To Ensure Efficacy.

*To Evaluate Benefit to Risk Ratio.

*To Collect additional data for Establishing

Physician Labeling.

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eCTD via Electronic Submission Gateway:

-FDA Form 356h

-FDA 3397: User Fee Cover Sheet

Payment of User Fees by PDUFA IV

$1,841,500 via Wire Transfer to U.S. bank in

St. Louis.

Submission of12 copies of Final Printed

Label with NDA.

Establishment:

-Risk Evaluation and Mitigation Strategies

-RiskMAPs

REMS Includes:

Medication Guide

Package Insert

Communication Plan for HCP’s.

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Post Marketing Plans:

Clinical Studies for Secondary Indications.

Pediatric Population

Launching Dosage Strengths

Free Patients Counseling

Promotional Campaign

Committed for Safety Reporting

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Thank You for Your

Attention

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