PDUFA on January 10 for tab-cel in EBV+ PTLD (priority review). If approved, it would be
the first approved treatment for this indication; ATRA would collect milestones and royalties from its
partner Pierre Fabre. Volatility around the decision is likely to be extreme.
FILSPARI is already approved in IgAN; the FSGS sNDA (PDUFA Jan 13 with AdCom) could
add a second meaningful leg. This is an institutionally crowded name with likely multiple moves
(pre-AdCom, post-AdCom, pre-PDUFA).
Anaphylm aims to change the anaphylaxis landscape with a film-based sublingual epinephrine.
PDUFA is set for January 31, 2026, with no AdCom. Retail interest is high and the “needle-free” angle
fuels a potential blockbuster narrative if efficacy and PK hold up.
Pediatric extension of leniolisib (Joenja) for APDS 4–11 years, PDUFA Jan 31 (priority review).
Joenja is already on the market in adults; pediatric approval would expand the addressable population
and reinforce Pharming’s profile as a revenue-generating rare-disease company.
With Nereus just approved for motion sickness, the market now has to price real peak sales,
launch velocity and optionality linked to GLP-1 side-effect management. Classic “post-approval price discovery”
setup with elevated volumes.
After the CRL on relacorilant in Cushing’s, the stock was cut in half. Korlym continues to generate cash
and the oncology program remains, but we’re firmly in “post-shock price discovery” territory:
large potential bounces, but with high regulatory uncertainty.
Another CRL for Lytenava in wet AMD. The “on-label Avastin for the eye” idea remains
intriguing, but the risk of further dilution and regulatory detours is high. This is a name for
news-driven trading, not for clean run-up plays.
Massive sell-off on setrusumab’s phase 3 failure in OI, but Ultragenyx still has a broad rare-disease
pipeline. Interesting to watch whether, after the initial shock, the market starts to re-price the
portfolio value beyond UX143.
Highly levered to setrusumab, Mereo was almost wiped out. Realistically, this belongs on the radar only
for potential special situations (asset sales, M&A, recap), with an extremely high risk profile.
D-MNA microneedle for BCC with phase 2 enrollment complete and topline data expected by end-Q1 2026.
Pure microcap setup: any clinical signal (good or bad) can drive outsized percentage moves.
Breakthrough designation for ulixacaltamide in essential tremor, with NDA planned
for early 2026. After spectacular post-data rallies, PRAX has become one of the most debated names
in XBI – with bulls pointing at ET as a huge unmet market and bears amplifying short-seller reports
on endpoint design and statistics.
BLA accepted for INO-3107 in RRP, with PDUFA set for October 30, 2026. Classic
“long runway run-up” candidate: the binary date is far away, but 2026 will bring multiple incremental
updates (FDA interactions, CMC, labeling discussions) that can serve as volatility waypoints.
Intranasal foralumab now has 37.4 patient-years of exposure without drug-related SAEs at a time when
the FDA has just rejected tolebrutinib (Sanofi) in nrSPMS on safety grounds. For CNS small-cap watchers,
TLSA is a “safety-first” case with multiple phase 2 readouts expected in 2026.