US FDA approval tracker: a first for Aimmune

January saw Aimmune receive an FDA approval for Palforzia, a first for peanut allergy, alongside decisions for Blueprint Medicines and Horizon Therapeutics.

The first month of 2020 saw the US FDA approve the first peanut allergy treatment, Aimmune’s Palforzia, greenlit after markets closed on Friday. The company's shares opened up 17% today, but quickly retreated to a 4% gain on concerns about Palforzia’s commercial opportunity.

The treatment has a black box warning for anaphylaxis and a restrictive risk-mitigation programme as it is based on a tolerising approach to peanut by the delivery of very small amounts of peanut powder in a capsule to the patient.

Questions remain over the long term benefits versus peanut avoidance, and in Palforzia’s clinical program patients on treatment had greater Epipen usage than those not on treatment, a consideration for payers given that this will come on top of the ~$10k annual cost of Palforzia.

EvaluatePharma's consensus puts 2024 sales at $1.3bn, a forecast that comprises some very different opinions; Stifel analysts, who are among the most conservative, believe that Palforzia will be become a "niche therapy". On the competitor front DBV’s desensitisation patch Viaskin Peanut has a PDUFA date in August.


Other positive decisions from the FDA last month included Blueprint’s Ayvakit, a month before its designated PDUFA date. Ayvakit is now approved for treating gastrointestinal stromal tumours driven by the PDGFRα exon 18 mutation, including D842V mutations. Expanding Ayvakit into a fourth-line setting will have to wait until the Voyager study reads out, due in the second quarter.

The decision on Horizon’s Tepezza was not expected until March, but the antibody became the first approved treatment for thyroid eye disease last month. The autoimmune condition leads to eye bulging and sometimes blindness. The disease can occur on its own but is also present in up to half of people with Graves’ disease.

The label language was broader than expected with no restrictions around disease type or severity, a topic that was heavily discussed at the unanimous ad com at the end of last year. The treatment has an annual list price of $200k and Horizon has guided to 2020 sales of $30-40m. 2024 sales are forecast to reach $484m according to EvaluatePharma consensus.

Notable first-time US approval decisions in January
Project Company 2024e sales ($m) Outcomes
Palfozia (AR101) Aimmune 1,312 Approved
Rimegepant ODT/Zydis ODT Biohaven 885 (rimegepant franchise) No decision yet, guided as Q1
Ayvakit (avapritinib)* Blueprint Medicines 673 Approved early
Tazverik (tazemetostat) Epizyme 556 Approved
Tepezza (teprotumumab)* Horizon Therapeutics 484 Approved early
Dificid (oral suspension) Merck/Astellas 168 (tablet and suspension) Approved
Travivo Fabre-Kramer Pharmaceuticals - No decision yet
Source: EvaluatePharma. *Not in January PDUFA story, source Go or no go? Enhertu’s destiny revealed and FDA decisions due for Epizyme, Novo, December 23, 2019.

And the rest

Novo Nordisk’s semaglutide was up for two decisions last month but only Ozempic, the once-weekly version, got its cardiovascular risk-reduction claim. Rybelsus, the oral, once-daily version, managed to get Pioneer 6 data added to its label, but that only showed non-inferiority to placebo. Rybelsus will have to wait for data from a long-term outcome study, Soul, which began earlier this year.

A decision on Biohaven’s Zydis ODT, a fast-acting dissolving tablet formulation of rimegepant for the acute treatment of migraine, is also still pending. More importantly the results of a phase III prevention study with the tablet formulation are expected soon, and could position rimegepant as a more convenient alternative to approved injectable CGRP inhibitors.

Supplementary and other notable approval decisions in January
Product Company Event type Outcomes
Keytruda  Merck & Co sBLA for high risk non-muscle invasive bladder cancer, with carcinoma in situ unresponsive to standard BCG therapy, based on Keynote-057 Approved
Ozempic and Rybelsus (oral semaglutide) Novo Nordisk sNDA for cardiovascular risk reduction Approved for Ozempic (Sustain 6Soul data needed for Rybelsus claim)
Dificid (tablet) Merck & Co/Astellas sNDA for Clostridium difficile infections in children aged six months or older, based on Sushine study Approved
Imfinzi Astrazeneca sBLA for first-line SCLC, based on Caspian trial No decision yet, guided as Q1
Mycamine Astellas sNDA for invasive candidiasis in paediatric patients under 4 months of age Approved
Source: Go or no go? Enhertu’s destiny revealed and FDA decisions due for Epizyme, Novo, December 23, 2019.

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