Every potential Food and Drug Administration (FDA) approval for a rare disease treatment gives hope for patients and physicians. So far in 2021, there are over 25 therapies focused on rare diseases that have  Prescription Drug User Fee Act (PDUFA) dates.

PDUFA dates refer to deadlines for the FDA to review new drugs.The FDA is typically given 10 months to review new drugs. If a drug is selected for priority review, the FDA is allotted 6 months to review the drug. These time frames begin on the date that an NDA is accepted by the FDA as complete. As each date approaches, CheckRare will be covering the FDA meetings and outcomes. We look forward to another exciting year in rare disease developments.

Orphan Drugs Currently Under Review by the FDA

Date

Orphan Drug Indication Company
January 23, 2021 Xalkori Pediatric ALK-positive anaplastic large cell lymphoma Pfizer
January 28, 2021 NPlate Hematopoietic syndrome of acute radiation syndrome Amgen
February 11, 2021 Evinacumab Homozygous familial hypercholesterolemia Regeneron
February 15, 2021 Umbralisib Marginal zone lymphoma TG Therapeutics
February 25, 2021 Casimersen Duchenne muscular dystrophy Sarepta Therapeutics
March 3, 2021 Libtayo Basal cell carcinoma Sanofi / Regeneron Pharmaceuticals
March 5, 2021 Axicabtagene ciloleucel Indolent B-cell non-Hodgkin lymphoma Gilead Sciences
March 27, 2021 Idecabtagene vicleucel Relapsed/refractory multiple myeloma bluebird bio / Bristol Myers Squibb
April 7, 2021 Brincidofovir Smallpox Chimerix
April 16, 2021 Treprostinil Pulmonary hypertension associated with interstitial lung disease United Therapeutics
April 23, 2021 Keytruda + platinum- and fluoropyrimidine-based chemotherapy Esophageal cancer Merck
April 27, 2021 Pegunigalsidase alfa Fabry disease Protalix BioTherapeutics
May 14, 2021 Pegcetacoplan Paroxysmal nocturnal hemoglobinuria Apellis Pharmaceuticals
May 18, 2021 Avalglucosidase alfa Pompe disease Sanofi
May 21, 2021 Loncastuximab tesirine Diffuse large B-cell lymphoma ADC Therapeutics
May 30, 2021 KD025 Chronic Graft-Versus-Host Disease Kadmon Holdings
June 1, 2021 Infigratinib

 

Cholangiocarcinoma BridgeBio Pharma
June 5, 2021 Plasminogen Plasminogen deficiency Liminal BioSciences
June 15, 2021 Umbralisib Follicular Lymphoma (FL) previously treated with at least 2 prior systemic therapies TG Therapeutics
June 15, 2021 TAK-721 Eosinophilic esophagitis Takeda
June 17, 2021 Arimoclomol Niemann-Pick disease Type C Orphazyme / CytRx Corp
June 25, 2021 Lonapegsomatropin Grown Hormone Deficiency in Children Ascendis Pharma
August 20, 2021 Vosoritide Achondroplasia BioMarin

 

Xalkori / Lymphoma

  • PDUFA Date: January 23, 2021
  • Company: Pfizer
  • Indicated for: Pediatric ALK-positive anaplastic large cell lymphoma
  • The supplemental New Drug Application (sNDA) is supported by results from Phase 1/2 Study ADVL0912and Phase 1 Study A8081013, which both showed compelling antitumor activity in pediatric and adult patients who received Xalkori.

 

NPlate / Radiation Poisoning

  • PDUFA Date: January 28, 2021
  • Company: Amgen
  • Indicated for: Hematopoietic syndrome of acute radiation syndrome
  • FDA submission likely based on positive results from a study demonstrating that romiplostim dramatically increases survival in mice following acute radiation. Romiplostim has been previously approved by the FDA for the treatment of immune thrombocytopenia.

 

Evinacumab (ANGPTL3 Antibody) / HoFH

  • PDUFA Date: February 11, 2021
  • Company: Regeneron Pharmaceuticals
  • Indicated for: Homozygous familial hypercholesterolemia (HoFH)
  • The Biologics Licence Application (BLA) is supported by the phase 3 ELIPSE HoFH trial which demonstrated that patients treated with evinacumab had a 47% reduction in low-density lipoprotein cholesterol compared with a 2% increase for patients treated with placebo.

 

Umbralisib / Marginal Zone Lymphoma

  • PDUFA Date: February 15, 2021
  • Company: TG Therapeutics
  • Indicated for: Marginal zone lymphoma (MZL) previously treated with at least 1 prior anti-CD20 based regimen
  • The NDA was supported by data from the umbralisib monotherapy MZL cohort of the UNITY-NHL Phase 2b trial.

 

Casimersen / Duchenne Muscular Dystrophy

  • PDUFA Date: February 25, 2021
  • Company: Sarepta Therapeutics
  • Indicated for: Duchenne muscular dystrophy
  • The NDA included data from the casimersen arm of the double-blind, placebo-controlled Phase 3 ESSENCE study. An interim analysis demonstrated a statistically significant increase in dystrophin production for patients receiving casimersen compared to baseline and placebo in patients amenable to exon 45 skipping.

 

Libtayo / Basal Cell Carcinoma

  • PDUFA Date: March 3, 2021
  • Company: Sanofi / Regeneron Pharmaceuticals
  • Indicated for: Basal cell carcinoma (BCC) patients who progress on or are intolerant to hedgehog inhibitors
  • The sBLA was based on data from a phase 2 clinical trial (NCT03132636) which demonstrated clinical benefit in patients with BCC.

 

Axicabtagene ciloleucel / Lymphoma

  • PDUFA Date: March 5, 2021
  • Company: Gilead Sciences
  • Indicated for: Indolent B-cell non-Hodgkin lymphoma
  • The sBLA was based on data from the primary analysis of the phase 2 ZUMA-5 trial in which 92% of patients receiving axicabtagene ciloleucel achieved an objective response to the treatment and 76% achieved a complete response.

 

Idecabtagene vicleucel / Multiple Myeloma

  • PDUFA Date: March 27, 2021
  • Company: bluebird bio / Bristol Myers Squibb
  • Indicated for: Relapsed/refractory multiple myeloma
  • The BLA was supported by positive data from the phase 2 KarMMa study. A presentation at ASH 2020concluded that clear and durable responses to idecabtagene vicleucel were observed, even in high-risk patients with relapsed or refractory multiple myeloma.

 

Oral brincidofovir / Smallpox

  • PDUFA Date: April 7, 2021
  • Company: Chimerix
  • Indicated for: Smallpox
  • According to a Chimerix press release, the NDA is partially based on two independent studies of the survival benefits of oral brincidofovir in animals infected with lethal animal models of smallpox.

 

Keytruda + platinum- and fluoropyrimidine-based chemotherapy / Esophageal Cancer

  • PDUFA Date: April 13, 2021
  • Company: Merck
  • Indicated for: Esophageal cancer
  • The sNDA is based on data from the phase 3 KEYNOTE-590 trial, in which Keytruda plus chemotherapy demonstrated significant improvements in overall survival and progression-free survival.

 

Treprostinil / Pulmonary Hypertension

  • PDUFA Date: April 16, 2021
  • Company: United Therapeutics
  • Indicated for: Pulmonary hypertension associated with interstitial lung disease (PH-ILD)
  • The sNDA is based on positive data from the phase 3 INCREASE clinical trial.

 

Pegunigalsidase alfa (PRX-102) / Fabry Disease

  • PDUFA Date: April 27, 2021
  • Company: Protalix BioTherapeutics
  • Indicated for: Fabry disease
  • The BLA is based on positive data from the phase 1/2 PRX-102 clinical trial and its extension study.

 

Pegcetacoplan / Paroxysmal Nocturnal Hemoglobinuria (PNH)

  • PDUFA Date: May 14, 2021
  • Company: Apellis Pharmaceuticals
  • Indicated for: Paroxysmal nocturnal hemoglobinuria (PNH)
  • The NDA is based on positive data from phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan to eculizumab.

 

Avalglucosidase alfa / Pompe Disease

  • PDUFA Date: May 18, 2021
  • Company: Sanofi
  • Indicated for: Pompe disease
  • The BLA is based on positive data from two trials: COMET and mini-COMET.

 

Loncastuximab tesirine / Lymphoma

  • PDUFA Date: May 21, 2021
  • Company: ADC Therapeutics
  • Indicated for: Diffuse large B-cell lymphoma
  • The BLA is based on data from the phase 3 LOTIS 2 trial, where loncastuximab tesirine induced an overall response rate of 48% and a complete response rate of 24%.

 

KD025 / Graft-Versus-Host Disease

  • PDUFA Date: May 30, 2021
  • Company: Kadmon Holdings
  • Indicated for: Chronic Graft-Versus-Host Disease (cGVHD)
  • The NDA is supported by positive data from the phase 2 KD025-213 trial, the results of which demonstrated that KD025 achieved a clinically meaningful objective response rate in patients with cGVHD.

 

Infigratinib / Cholangiocarcinoma

  • PDUFA Date: June 1, 2021
  • Company: BridgeBio Pharma
  • Indicated for: Cholangiocarcinoma
  • The NDA is supported by results of a phase 2, multicenter, single arm study (NCT02150967) of infigratinib as a third- or later-line treatment of patients with advanced cholangiocarcinoma.

 

Ryplazim (plasminogen) / Congenital Plasminogen Deficiency

  • PDUFA Date: June 5, 2021
  • Company: Liminal BioSciences
  • Indicated for: Congenital Plasminogen Deficiency (C-PLGD)
  • The BLA is supported by data from a phase 2/3 clinical trial which evaluated both adult and pediatric patients with C-PLGD.

 

Umbralisib (UNITY-NHL) / Follicular Lymphoma

  • PDUFA Date: June 15, 2021
  • Company: TG Therapeutics
  • Indicated for: Follicular Lymphoma (FL) previously treated with at least 2 prior systemic therapies
  • The NDA was primarily based on data from the umbralisib monotherapy FL cohort of the UNITY-NHLPhase 2b trial. The primary endpoint of overall response rate (ORR) was met for all treated FL patients.

 

TAK-721 / Eosinophilic Esophagitis

  • PDUFA Date: June 15, 2021
  • Company: Takeda
  • Indicated for: Eosinophilic Esophagitis
  • The NDA designation is supported by positive data from a phase 3 study evaluating TAK-721 in adolescent and adult patients with eosinophilic esophagitis.

 

Arimoclomol / Niemann-Pick Disease Type C

  • PDUFA Date: June 17, 2021
  • Company: Orphazyme / CytRx Corp.
  • Indicated for: Niemann-Pick Disease Type C (NPC)
  • The NDA designation is supported by positive data from a phase 2/3 study evaluating the safety and efficacy of arimoclomol compared to placebo as an add-on therapy.

 

Lonapegsomatropin / Grown Hormone Deficiency

  • PDUFA Date: June 25, 2021
  • Company: Ascendis Pharma
  • Indicated for: Grown Hormone Deficiency in Children
  • The BLA designation is supported by the results of a clinical development program that included eight clinical trials evaluating the safety and efficacy of lonapegsomatropin in GHD patients, according to an Ascendis Pharma press release.

 

Vosoritide / Achondroplasia

  • PDUFA Date: August 20, 2021
  • Company: BioMarin
  • Indicated for: Achondroplasia
  • The NDA designation is supported by positive results from a 1-year phase 3 trial and a 5-year follow up phase 2 trial.