Pharmaceutical nanotechnology has generated breakthrough developments in improving health care and human life from its emergence. The biomaterials employed mainly aim at improving drug delivery systems, imaging and diagnostic technologies while the nanoscale materials are in widespread use in other industries such as electronics and optics. Such advancement may revolutionize the drug development and therapy with new and more efficient treatments. Although, nanotechnology assists humankind in improving its well being, it has certain limitations that entail thorough investigation by the regulatory and scientific authorities. To address concerns regarding the safety and toxicity profile of the nanopharmaceuticals, we have reviewed the challenges and solutions of nanopharmaceuticals use in human health and the related health risks. In this regard, regulatory and scientific bodies such as countries’ Food and Drug Administration (FDA), Organization for Economic Co-operation and Development (OECD), European Medicine Agency (EMA), Environmental Protection Agency (EPA), National Institute for Occupational Safety and Health (NIOSH), and World Health Organization (WHO) can participate in developing and reinforcing safety measures and regulatory frameworks to insure the public health. The regulatory authorities may enforce the nanopharmaceutical industries to conduct comprehensive toxicity tests and monitor the adverse drug reaction reports in close collaboration with the scientific community to act accordingly and inform the public as the implementation of the strategy.Graphical abstractNanopharmaceuticals have tremendous potential for human use as therapeutic or diagnostic agents. But their toxicity profile should be well addressed and the respective regulatory framework developed and reinforced by the authorities.