Shots:
The EC approved Aspaveli (pegcetacoplan) for pts (≥12yrs.) with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), to be used with a RAS inhibitor unless such therapy is contraindicated or not tolerated
Approval was based on P-III (VALIANT) trial assessing Aspaveli(twice weekly) vs PBO in C3G or primary IC-MPGN pts (≥12yrs., n=124) for…
Shots:
Sobi has entered into a definitive agreement to acquire Arthrosi Therapeutics, incl. its asset pozdeutinurad (AR882), expanding its gout pipeline
As per the deal, Sobi will acquire Arthrosi in an all-cash transaction for $950M upfront & ~$550M in clinical, regulatory & sales milestones; closing is expected in H1’26
Pozdeutinurad (QD, PO) is an URAT1…
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The US FDA has accepted the BLA of Nanoecapsulated Sirolimus + Pegadricase (NASP; Q4W) for the treatment of uncontrolled gout (PDUFA: Jun 27, 2026)
BLA was supported by P-III (DISSOLVE I & II) trials evaluating 2 NASP doses vs PBO in adults with uncontrolled gout, which met its 1EP of sustained decrease in serum…
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The US FDA has approved Doptelet to treat thrombocytopenia in pts (≥1yrs.) with persistent or chronic ITP who have not responded to prior treatments. Approval also incl. a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for children (1-6yrs.)
Approval was based on P-III (AVA-PED-301) study assessing Doptelet in pediatric ITP pts, which showed that…
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The US FDA has approved Gamifant (emapalumab-lzsg) for treating hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still's disease, including systemic Juvenile Idiopathic Arthritis (sJIA), in pts of all ages who are unresponsive, intolerant to glucocorticoids, or have recurrent MAS
Approval was based on pooled data from two pivotal trials, P-III (EMERALD) and P-II (NI-0501-06),…
Shots:
Apellis & Sobi have reported data from the open-label period of P-III (VALIANT) trial of Empaveli for the treatment of C3G & primary immune complex membranoproliferative glomerulonephritis (IC-MPGN); regulatory review is ongoing in the US & EU
The P-III (VALIANT) trial assessed Empaveli (Q2W) vs PBO in C3G or primary IC-MPGN pts (≥12yrs., n=124)…
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Ionis has granted Sobi global exclusive rights of olezarsen excl. the US, Canada & China to treat familial chylomicronemia syndrome (FCS) & severely elevated triglycerides; Ionis to independently market Olezarsen in the US, whereas Theratechnologies holds the rights in Canada
As per the deal, Ionis will get an upfront, with potential milestone payments &…
Shots:
The second quarter of 2023 highlights multiple clinical trial results, approvals, and numerous deals. There are major alliances in this quarter which include Merck acquired Prometheus for ~$10.8B and Astellas entered into a definitive agreement to acquire Iveric Bio for ~$5.9B
The second quarter of the year also showcases multiple approvals of the therapies…
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The EMA approved 3 New Chemical Entity (NCE) and 7 Biologic Drugs in May 2023, leading to treatments for patients and advances in the healthcare industry
In May 2023, the major highlights drugs were Breyanzi’s Approval for relapsed or refractory large B-cell lymphoma, Ultomiris (ravulizumab) for neuromyelitis optica spectrum disorder
PharmaShots has compiled a…
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The first quarter of 2023 highlights major acquisitions in the pharma and biotech industry along with multiple approvals. Meanwhile, Selecta and Sobi reported P-III trial (DISSOLVE I & II) results of SEL-212 for chronic refractory gout
Starting with the latest acquisitions, Pfizer acquired Seagen for ~$43B. The first quarter of the year also showcases…

