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A Busy Last Week of April for PDUFAs!


  • As we are almost in the last week of April, many PDUFA decisions are still to come!

  • Next week will be busy for PDUFAs, with 6 decisions still pending

  • What other assets have similar MoAs that will be competitors to these possibly approved products?

  • Also see which May PDUFA already reached a decision!

See our previous free forum with additional info on historical PDUFA decisions since September 2021!!

About PDUFA dates An important note to remember especially for newer investors is that PDUFA target dates are just that, a target date. Thus, approval usually happens just before or on the date. However, especially with Covid travel restrictions affecting site inspections, approval or a CRL (i.e. a letter from FDA that the drug candidate will NOT be approved unless something is fixed), can come after the target date.

IONS - Tofersen PDUFA 4/25/23

  • Mechanism of action

    • Tofersen is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS). There is substantial evidence that mutations in the SOD1 gene are responsible for a toxic gain of function that can lead to rapid progressive loss of motor neurons in patients with SOD1-ALS.

  • Competitors to Tofersen

    • Currently there is no cure for ALS, but the approved drug riluzole is approved for treatment to slow the progression of the disease

    • Tofersen would be approved for patients with the SOD1-ALS gene mutation, which includes ALS patients that can accumulate toxic levels of the protein causing neuron degeneration

    • Competitors: CLNN CNM-Au8 (Phase 3), CYTK Reldesemtiv (Phase 3), and SEEL SLS-005 (Phase 2/3)

    • CNM-Au8 could help protect patients' nerves against oxidative stress, cellular damage found in ALS patients

    • Reldesemtiv could slow the rate of calcium release for patients, leading to an increase in muscle contractility and respond to weak nerve signals

    • SLS-005 could promote the clearance TDP-43 and SOD1 proteins, the accumulation of which can be toxic to nerve cells

  • Market

    • The market for ALS in 2018 was $537M and is expected to grow 5.8% by 2026

    • However the treatment is typically costly

  • Other assets from company

    • IONS has one approved product (SPINRAZA for spinal muscular atrophy) as well as a second product with a PDUFA scheduled for the end of the year (Eplontersen for transthyretin amyloidosis)

    • IONS has many other assets in Phase 2b or Phase 3 trials


  • Mechanism of action

    • Trikafta is a CFTR modulator that helps defective CFTR proteins work more effectively. Both elexacaftor and tezacaftor work as correctors. They bind to the faulty CFTR protein and help it fold correctly. This way, cells can shuttle more of it to the membrane instead of degrading the newly formed protein

    • Other CFTR assets for cystic fibrosis include FDMT 4D-710 and ARCT ARCT-032. The VRNA asset ensifentrine treats COPD and is also a CFTR modulator

  • Competitors and stages

    • Currently treatments include methods to ease symptoms and reduce complications including antibiotics, cough medicine and penicillin, a chest wall oscillation procedure, and supportive care with sodium chloride

    • Competitors: ARMP AP-PA02 (Phase 1/2), FDMT 4D-710 (Phase 1/2), and ARCT ARCT-032 (Phasew 1)

    • AP-PA02 is a therapeutic cocktail that targets a pathogen to treat respiratory infections, especially in patients with cystic fibrosis

    • 4D-710 is a targeted and evelved vector to treat a variety of patients, independent of the CFTR mutation and designed to achieve CFTR expression

    • TRIKAFTA is already approved, but this would expand the label to include children as young as 2 years old

  • Market

    • Current in 2022 the market size is $5.3B and is projected to grow to $12.9B by 2030

  • Other assets from company

    • VRTX has a second cystic fibrosis asset with a PDUFA in May 2023. Also are othe Phase 2 and Phase 3 assets in rare diseases

ASND TransCon PTH 4/30/23

  • Mechanism of action

    • TransCon PTH contains a long-lasting precursor of PTH. The therapy is a prodrug — an inactive compound converted by the body into an active therapy — designed to be administered once daily via under-the-skin (subcutaneous) injection to normalize PTH levels

  • Competitors and stages

    • Currently treatments for hypoparathyroidism include activated vitamin D and calcium supplements.

    • Competitors: XERS Levothyroxine (Phase 2)

    • Levothyroxine is administered when the body is deficient in the endogenous hormone. Administration of levothyroxine is thus indicated for acquired thyroid disease (primary hypothyroidism), in cases of decreased secretion of TSH from the anterior pituitary gland (secondary hypothyroidism), and in cases of decreased secretion of TRH from the hypothalamus (tertiary hypothyroidism) and for congenital hypothyroidism.

  • Market

    • The market for hypoparathyroidism is expected to reach $1.3B by 2026

  • Other assetds from company

    • ASND currently has one approved asset for pediatric growth hormone deficiency and multiple Phase 2 and Phase 3 assets also in growth disorders

GMDA Omidubicel 5/1/2023

  • Omidubicel was approved early on 4/17/2023 and stock jumped almost 50%!

  • 11 PDUFAs are coming in May

Sign up for free to see historical PDUFA info!

This article is not investment, tax, or legal advice. Please do your own diligence and seek advice from professional advisors representing your interests.

Article history:

Originally posted 4/18/23


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