Drugs Approved by FDA Despite Failed Trials or Minimal/Insufficient Data

I have been trying to collect the cases of that drugs were approved by the FDA despite the failed trials or minimal/insufficient data. For drugs treating rare diseases or diseases with unmet medical needs, the FDA may apply flexibility in approving the drug with loosened criteria. 

For diseases with clearly unmet medical needs such as ALS (Amyotrophic Lateral Sclerosis) and Alzheimer's disease, FDA officials have recently emphasized the urgent need for new treatments and pledged to use maximum "regulatory flexibility" when reviewing the NDA/BLA packages. By applying the maximum "regulatory flexibility", FDA has approved some drugs which do not meet the agency's traditional approval standards. Some of the approvals are really controversial and make me wonder if there is any boundary for the maximum "regulatory flexibility". 

The following paper on BioSpace.com listed six drugs that earned FDA approval without substantial evidence of effectiveness.

6 Drugs Approved Despite Failed Trials or Minimal Data

• Ipsen’s Sohonos (palovarotene) for the ultra-rare genetic disease fibrodysplasia ossificans progressive (FOP)
• Sarepta’s Elevidys as the first gene therapy for Duchenne muscular dystrophy (DMD)
• Biogen's Qalsody (tofersen) to treat patients with superoxide dismutase 1 (SOD1)-ALS, a rare subtype of the fatal neurodegenerative disease
• Biogen and Eisai got the nod for Aduhelm (aducanumab) for Alzheimer's diease,
• Jazz Pharmaceuticals and PharmaMar’s Zepzelca (lurbinectedin) for small cell lung cancer (SCLC) that had progressed on or after platinum-based chemotherapy
• Acadia Pharmaceuticals’ Nuplazid (pimavanserin) to treat hallucinations and delusions associated with psychosis in Parkinson’s disease.

Some of the approvals gave the sponsors the false hope that an innovative drug could be approved by the FDA even if the study failed to demonstrate the effectiveness as long as the drug was for the treatment of diseases with urgent unmet medical needs. A recent story about BrainStorm's ALS drug is exactly the case about this.