Rare disease

Italy’s Angelini pays $4.1B cash for rare disease specialist Catalyst

Thu, 07 May 2026 12:00:00 -0400

Italian family-owned Angelini Pharma SpA is making its first move into the U.S. market, acquiring rare diseases specialist Catalyst Pharma Inc. in an all-cash deal worth $4.1 billion. The acquisition gives Angelini ownership of three marketed drugs for treating epilepsy and neuromuscular diseases that had combined sales of $589 million in 2025, a 19.8% increase over 2024.

Up to BAT in PSC, Mirum base case proven with phase IIb

Mon, 04 May 2026 12:00:00 -0400

Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).

Lilly buying Ajax for up to $2.3B for next-gen JAK assets

Mon, 27 Apr 2026 12:00:00 -0400

Eli Lilly and Co. is acquiring Ajax Therapeutics Inc. for up to $2.3 billion in cash, gaining access to next-generation JAK inhibitors for patients with myeloproliferative neoplasms.

Travere’s Filspari approved for rare kidney disease FSGS

Tue, 14 Apr 2026 08:00:00 -0400

Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint.

Synox en route to approval on positive phase III for TGCT

Mon, 13 Apr 2026 12:00:00 -0400

Synox Therapeutics Ltd. is preparing to file for FDA approval of emactuzumab in treating tenosynovial giant cell tumor (TGCT), after announcing positive top-line phase III results.

Pediatric PRVs top the voucher popularity contest

Thu, 09 Apr 2026 12:00:00 -0400

With three rare pediatric disease priority review vouchers (RPD PRVs) awarded just since the end of March, the nearly year-and-a-half lapse in the program’s reauthorization seems to have had little short-term impact. The three new vouchers bring the total RPD PRVs granted so far this year to seven – one more than the agency issued all last year and down two from the nine given in 2024.

Vykat caveat: Neurocrine’s $2.9B Soleno bid low-ball?

Mon, 06 Apr 2026 12:00:00 -0400

A proposal to buy out Soleno Therapeutics Inc. didn’t wait for European approval of Vykat XR (diazoxide choline) to treat hyperphagia in Prader-Willi syndrome, as Neurocrine Biosciences Inc. is putting on the table $53 per share in cash, which equates to an equity value of $2.9 billion.

LHON updates: Gene therapy progress; idebenone receives US CRL

Thu, 02 Apr 2026 12:00:00 -0400

Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

FDA approves Rocket’s Kresladi as first gene therapy for LAD-I

Fri, 27 Mar 2026 12:00:00 -0400

In a win for the rare disease space, the U.S. FDA granted accelerated approval for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel) as the first gene therapy option for treating severe leukocyte adhesion deficiency-I (LAD-I), an ultrarare genetic immune disorder characterized by an immunodeficiency predisposing those affected to recurrent and fatal infections.

GSK’s IBAT inhibitor wins US FDA nod for cholestatic pruritus

Thu, 19 Mar 2026 12:00:00 -0400

With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus.

Pharma USA: The complex journey of launching rare disease drugs

Tue, 17 Mar 2026 12:00:00 -0400

Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients.

Forecast brighter for Uniqure with Prasad’s departure?

Mon, 09 Mar 2026 12:00:00 -0400

The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay Prasad as director of the agency’s CBER at the end of April.

FDA’s rare disease toolbox not fully used

Wed, 04 Mar 2026 12:00:00 -0500

At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.

Experts: Regulatory roadblocks stalling rare disease therapies

Tue, 03 Mar 2026 12:00:00 -0500

A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.

Theravance slashes R&D as hypotension asset fails again

Tue, 03 Mar 2026 12:00:00 -0500

Another phase III failure of Theravance Biopharma Inc.’s norepinephrine reuptake inhibitor ampreloxetine to treat symptomatic neurogenic orthostatic hypotension, this time in patients with the rare disease multiple system atrophy, means the end of the company’s R&D efforts. Cutting its workforce in half and terminating all development of ampreloxetine, its only pipeline product, the Dublin-based company’s shares (NASDAQ:TBPH) sank by 26%, or $4.99, to close March 3 at $13.96.

State of the biopharma industry filled with uncertainty

Wed, 25 Feb 2026 12:00:00 -0500

While the annual State of the Union address has morphed over the years from a summation of the state of the U.S. government and the president’s legislative agenda into political theater on both sides of the aisle, President Donald Trump did include some recommendations to Congress in his Feb. 24 speech. Among those recommendations was a request for Congress to codify his most-favored-nation pricing policy for prescription drugs.

New FDA guidance details ‘plausible mechanism’ pathway

Mon, 23 Feb 2026 12:00:00 -0500

The U.S. FDA rolled out its anticipated “plausible mechanism” draft guidance, enabling ultrarare disease drug developers to generate evidence of effectiveness and safety to support approval when randomized controlled trials are not possible due to small patient populations.

Intellia’s nex-z moves ahead, but only for ATTR-PN for now

Tue, 27 Jan 2026 12:00:00 -0500

Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.

Tanabe’s dersimelagon hits phase III endpoint in rare disorder

Tue, 27 Jan 2026 00:00:00 -0500

After selling off amyotrophic lateral sclerosis therapeutics last month, Tanabe Pharma America Inc. emerged a forerunner in another rare disease space with positive top-line phase III data of dersimelagon (MT-7117) for erythropoietic protoporphyria/X-linked protoporphyria.

NIH spared slash-and-burn in congressional spending agreement

Wed, 21 Jan 2026 12:00:00 -0500

The good news is that the U.S. Congress is on track to pass a slate of fiscal 2026 spending bills before the current continuing resolution expires Jan. 30. So, barring any last-minute disputes or legislative hostage-taking, there should be no repeat of last year’s 43-day shutdown that impacted NIH grants and activities.

Tanabe’s dersimelagon hits phase III endpoint in rare disorder

Wed, 21 Jan 2026 12:00:00 -0500

US FDA clears Sentynl’s Zycubo as first treatment for Menkes

Tue, 13 Jan 2026 12:00:00 -0500

The U.S. FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease, a rare, genetic disease affecting children who cannot absorb copper through their intestines, leading to seizures, weak muscles, a failure to thrive and, ultimately, if left untreated, an early death by age 3.

Atara, Pierre Fabre: FDA flipflops on Ebvallo’s rare disease BLA

Mon, 12 Jan 2026 12:00:00 -0500

A second complete response letter issued by the U.S. FDA for Atara Biotherapeutics Inc.’s allogeneic T-cell immunotherapy Ebvallo (tabelecleucel) for Epstein-Barr virus positive post-transplant lymphoproliferative disease is “a complete reversal of position” by the agency, which had previously confirmed the single arm Allele trial was enough to support a BLA under the accelerated approval pathway, the company said.

FDA takes flexibility, modernization steps in CGT, Rx development

Mon, 12 Jan 2026 12:00:00 -0500

Moving away from a one-size-fits-all approach, the U.S. FDA's CBER released details Jan. 11 about how it’s leveraging its growing experience with cell and gene therapies (CGTs) to exercise greater regulatory flexibility in chemical, manufacturing and control requirements for the products.

Drugs to Watch 2026: 11 therapies that could transform patient care

Tue, 06 Jan 2026 12:00:00 -0500

The newly released 2026 edition of Clarivate’s Drugs to Watch report highlights 11 potential blockbusters that could change treatment paradigms for patients.

2025 marks a breakthrough year for in vivo gene therapies

Tue, 30 Dec 2025 08:00:00 -0500

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them.

2025 marks a breakthrough year for in vivo gene therapies

Tue, 30 Dec 2025 08:00:00 -0500

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.

2025 marks a breakthrough year for in vivo gene therapies

Mon, 29 Dec 2025 08:00:00 -0500

US Senate ends 2025 with no gift for kids with cancer

Tue, 23 Dec 2025 12:00:00 -0500

After being unanimously passed by the U.S. House Dec. 1, the bipartisan Mikaela Naylon Give Kids a Chance Act seemed to be headed for sure passage in the Senate before it adjourned late last week.

Shionogi nabs revenue-generating ALS drugs with $2.5B Tanabe pact

Tue, 23 Dec 2025 12:00:00 -0500

Shionogi & Co. Ltd. will acquire global rights to U.S. FDA approved amyotrophic lateral sclerosis therapy edaravone through a $2.5 billion acquisition deal with Tanabe Pharma Corp. Under the terms, Tanabe will form a new entity harboring both oral and intravenous (I.V.) infusion formulations of edaravone that are marketed in the U.S. as Radicava ORS and I.V. Radicava.