The biggest regulatory event of the year happens next month, when Biogen’s Alzheimer’s candidate aducanumab receives a final verdict from the FDA. While the US regulator has been enthusiastic about aducanumab the project’s advisory committee meeting was overwhelmingly negative, making a green light far from certain.
One June approval that jumped the gun was that of Myovant's relugolix combination Myfembree, for uterine fibroids. This had been due on June 1 but came yesterday. The FDA still has a busy month ahead: Ascendis could gain its first approval with the growth hormone asset Transcon hGH, and Novo Nordisk has obesity in its sights with semaglutide.
But June really is all about aducanumab, and for the MAb to be approved the FDA would have to rule against its own adcom, an outcome that is rare but not unknown. The most damning vote at the meeting was 10-0 against on the question of whether the Emerge trial could be considered primary evidence of the project’s effectiveness.
Emerge, along with adu's other pivotal trial, Engage, failed at interim analyses, but Biogen later said longer-term data showed a hit in the former (Shock revelation sees Biogen erase its aducanumab losses, October 22, 2019). The FDA has been supportive, and given the lack of Alzheimer's treatment options there is pressure to approve.
The outcome will be relevant for other companies with beta-amyloid MAbs in development; Roche and Lilly both have several in clinical trials, while Biogen itself also has a second MAb, BAN2401, in phase 3.
Meanwhile, Ascendis could see its first US approval next month with Transcon hGh, a long-acting once-weekly human growth hormone for paediatric growth hormone deficiency.
Transcon hGh has a shot at being market leader in the space by 2026, overtaking Novo’s once-daily growth hormone norditropin – if it can get a superiority claim on its label. Phase 3 data showed greater efficacy than daily injections (Ascendis shoots higher on pivotal win, March 4, 2019). Consensus forecasts, as compiled by Evaluate Pharma, see Transcon hGh sales of $1.4bn by 2026.
Other once-weekly projects to watch include Pfizer/Opko’s somatrogon, which has a Pdufa in October, although it has so far only shown non-inferiority to daily injections (Pfizer’s growth hormone falls short, October 22, 2019). Novo’s once-weekly candidate somapacitan is further behind, with data from a paediatric phase 3 study expected in the second half.
Pegged for the second quarter, approval of Novo’s semaglutide in obesity looks to be in the bag after impressive data from the Step programme. Novo had been targeting non-placebo-adjusted weight loss of at least 12%, while clinical trials found 17-18% (Novo adds weight to its obesity goals, September 10, 2020).
Sellside consensus forecasts from EvaluatePharma see $2.4bn for sema in obesity by 2026, outdoing Novo’s own existing obesity drug Saxenda, which has only shown around 5-7% weight loss. Semaglutide is already marketed for diabetes as Ozempic, an injectable, and Rybelsus, a pill.
The tables below list first-time and supplementary US approvals and advisory meetings due next month, with consensus forecasts from Evaluate Pharma.
|Notable first-time US approval decisions due in June|
|Project||Company||PDUFA date||Indication(s)||2026e sales by indication ($m)||Note|
|PF-06482077||Pfizer||June||Pneumonia||1,037||20-valent pneumococcal conjugate vaccine|
|Alkermes||June 1 (resubmission)||Schizophrenia and bipolar I disorder||380||CRL last year over CMC issues|
|Brexafemme||Scynexis||June 1||Vulvovaginal candidiasis||302||-|
|Infigratinib||Bridgebio||Est June 1||2L cholangiocarcinoma||253||FGFR inhibitor|
|Ryplazim||Liminal||June 5 (resubmission)||Congenital plasminogen deficiency||65||CRL in 2017 over CMC issues|
|Aducanumab||Biogen/Eisai||June 7||Alzheimer's disease||2,396||See text|
|Miplyffa (arimoclomol)||Orphazyme||June 17||Niemann Pick disease type C||293||Extended from March|
|Molluscum contagiosum||271||CRL in 2020 over CMC issues|
|Transcon hGH||Ascendis||June 25||Paediatric growth hormone deficiency||1,388||See text|
|Verkazia||Santen||June 26||Keratoconjunctivitis (patients aged 4-18)||-||Topical ophthalmic cyclosporine|
|June 29||Severe thermal burns||50||Mediwound could feel the burn with Nexobrid launch|
|Takeda||June?||Eosinophilic oesophagitis||341||Pdufa was in Apr but no decision has been made|
|IV Tramadol||Avenue||June?||Acute pain||263||Pdufa was in Apr but no decision has been made|
|StrataGraft||Mallinckrodt||June?||Adults with deep partial-thickness thermal burns||59||Action was deferred in Feb owing to travel restrictions, no new date given|
|Tanezumab||Pfizer/Lilly||June?||Moderate-severe osteoarthritis pain||63||Negative adcom in Mar, no new Pdufa date set|
|Source: Evaluate Pharma & company releases.|
|Advisory committee meetings in June|
|Project||Company||Adcom date||Indication||2026e sales by indication ($m)||Note|
|Covid-19 vaccines||-||June 10||Prevention of Covid-19||-||To discuss data needed to support the use of Covid-19 vaccines in children less than 12 years of age. The committee will not discuss any specific products.|
|Incyte/Macrogenics||June 24||Adults with locally advanced or met squamous carcinoma of the anal canal who have progressed on or who are intolerant of platinum chemo||53||Anti-PD-1 MAb|
|Source: Evaluate Pharma & FDA adcom calendar.|
|Supplementary and other notable approval decisions in June|
|Product||Company||Indication (clinical trial)||Date|
|Cosentyx||Novartis||Moderate-severe plaque psoriasis in children and adolescents (NCT03668613, NCT02471144)||Est June|
|Rinvoq||Abbvie||Psoriatic arthritis (Select-PsA 1, Select-PsA 2)||June|
|Ankylosing spondylitis (Select-Axis 1)|
|Ultomiris||Alexion/ Astrazeneca||Children and adolescents with PNH||June 7|
|Trikafta||Vertex||Children aged 6 to 11 who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive (NCT03691779)||June 8|
|Ayvakit||Blueprint||Advanced systemic mastocytosis (Pathfinder, Explorer)||June 16|
|Ruxolitinib cream||Incyte||Atopic dermatitis (TruE-AD1, TruE-AD2)||June 21|
|Jakafi||Incyte/Novartis||Paediatric patients with steroid-refractory GvHD (Reach3)||June 22|
|Solosec||Lupin||Treatment of trichomoniasis in adults and adolescents||June 30|
|Nurtec ODT||Biohaven||Migraine prevention (Study 305, 201)||Q2|
|Alecensa||Roche||1st-line Alk-positive NSCLC (Bfast)||Q2|
|Semaglutide 2.4mg (Ozempic)||Novo Nordisk||Obesity (Step 1, 2, 3 and 4)||Q2|
|Source: Evaluate Pharma & company releases.|
This article has been updated to include the June panel meeting to discuss paediatric use of Covid-19 vaccines.